Clinical Trials

Title Sponsor Keywords Start Date Completion Date
iCanCope With Sickle Cell PainSeattle Children's HospitalJan 2018Dec 2022
A Phase - IIa - IIb, Open Label Trial to Study the Safety, Tolerability and Efficacy of Memantine as a Long-term Treatment of SCDHaEmek Medical Center, IsraelJan 2018Jun 2020
A Study of the Effect of IW-1701, a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)Ironwood Pharmaceuticals, Inc.Sickle Cell Disease, SCDDec 2017Jul 2019
Non-Myeloablative Conditioning Regimen With Haploidentical T-Cell-Depleted Peripheral Blood Transplant for Patients With Severe Sickle Cell DiseaseCity of Hope Medical CenterSickle Cell Disease, Sickle Cell Disorders, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell, Haploidentical Transplant, Nonmyeloablative Conditioning, CD4+ T cell, CD4+ T cell-depleted Hematopoietic Cell Transplant, Mixed ChimerismNov 2017Nov 2021
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent Beta-ThalassemiaHoffmann-La RocheOct 2017May 2018
Sickle Cell Disease: Targeting Alloantibody Formation Reduction; Risk Factors, and GeneticsSanquin Research & Blood Bank DivisionsOct 2017Dec 2019
Enhancing Preventive Therapy of Malaria In Children With Sickle Cell Anemia in East Africa (EPiTOMISE)Duke UniversityOct 2017Oct 2019
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell DiseaseCity of Hope Medical CenterSickle Cell Disease, Hematopoietic stem cell transplantation, Haploidentical stem cell transplantation, Post-transplant CytoxanOct 2017Aug 2022
Gene Transfer for Sickle Cell DiseaseDavid WilliamsGene therapy, lentivirus vector, BCL11A, Fetal HemoglobinOct 2017Oct 2024
Muscle Function and Its Biological and Physiological Determinants in Sickle Cell DiseaseHospices Civils de LyonSickle Cell Disease, Muscle function, Hemorheological disordersSep 2017Sep 2019
Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically ModifiedNanfang Hospital of Southern Medical UniversityBeta thalassemia, Gene therapy, Beta-globin, Hematopoietic stem cells, Lentiviral vectorSep 2017Sep 2021
iCanCope With Sickle Cell Disease: a Computerized Cognitive Behavioral Therapy Program for Pain ManagementUniversity of PittsburghSep 2017Sep 2019
Adiponectin, IL-6 and hsC-RP in Relation to Carotid Intima-media Thickness in B-thalassemia PatientsAsmaa Nady HusseinSep 2017Mar 2020
HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell DiseaseCentre Hospitalier Intercommunal Creteilsickle cell disease, haploidentical, graft, marrowSep 2017Sep 2020
sPLA2 in EBC During Acute Chest SyndromeVirginia Commonwealth Universityacute chest syndrome, secretory phospholipases A2, exhaled breath condensate, sickle cell diseaseSep 2017Feb 2018
Haploidentical Bone Marrow Transplantation in Sickle Cell PatientsMedical College of WisconsinReduced Intensity Conditioning, Haploidentical, Bone Marrow, Transplant, Sickle Cell DiseaseSep 2017Dec 2024
Kidney Function in Sickle Cell AnemiaUniversity of North Carolina, Chapel HillSep 2017Aug 2022
Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell DiseaseEmory UniversityHematopoietic cell transplantation (HCT), Haploidentical, Mesenchymal stromal cellsSep 2017Sep 2021
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCTPhoenix Children's HospitalAug 2017Oct 2018
Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) PatientsNational Heart, Lung, and Blood Institute (NHLBI)Plerixafor, Leukapheresis, Mobilization, Stem Cells, Sickle Cell DiseaseJul 2017Feb 2027
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling DonorUniversity of Calgarysickle cell disease, stem cell transplant, red blood cell engraftment, nonmyeloablative, pure red cell aplasiaJul 2017Jul 2023
Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease and Healthy ControlsUniversity of California, Los AngelesAdolescence, Autonomic Nervous System, Hypnosis, Pain, Sickle Cell DiseaseJul 2017Jun 2020
BEATS 2: Music Therapy in Sickle CellUniversity Hospitals Cleveland Medical CenterJun 2017Dec 2018
An Open Label Randomized Controlled Trial to Evaluate the Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi PakistanDr.Saqib Hussain AnsariJun 2017Jan 2018
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersIonis Pharmaceuticals, Inc.Thalassaemia, IONIS TMPRSS6-LrxJun 2017Mar 2018
Erythrocyte Glutamine/Glutamate Ratio Relation to Pulmonary Hypertension Risk in Thalassemic ChildrenAssiut UniversityThalassemia, Glutamine/glutamate ratio, Pulmonary hypertensionJun 2017Jun 2018
Sleep and Pain in Sickle Cell DiseaseJohns Hopkins UniversityJun 2017May 2021
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children HospitalAssiut Universitythalassemia - nutrition -growthJun 2017Dec 2018
Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical CareNational Human Genome Research Institute (NHGRI)CRISPR, Gene Editing, Qualitative, Participation Clinical Trials, Genetic LiteracyJun 2017Jun 2018
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotypebluebird bioJun 2017Apr 2021
A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered.AstraZenecaAbnormal hemoglobin (called hemoglobin S or sickle hemoglobin) in the red blood cells, Thrombotic cardiovascular events, Acute coronary syndrome, P2Y12 platelet inhibitor, BrilintaMay 2017Jul 2017
The Effective and Safety of Thalidomide in TIXiao-Lin YinThalidomide TIMay 2017May 2019
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell DiseaseIncyte CorporationSickle cell disease (SCD), sickle cell SS, lysine demethylase 1 (LSD1) inhibitionMay 2017Jun 2017
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell DiseaseUniversity of Illinois at ChicagoApr 2017Oct 2026
Denosumab Versus Zoledronic Acid in Thalassemia-Induced OsteoporosisHamad Medical CorporationOsteoporosis, Beta-Thalassemia Major, Denosumab, Zoledronic AcidApr 2017Apr 2018
EPO-4-Rhesus: Role of Darbepoetin Alfa in Reducing Late Anaemia in Neonates With Red Blood Cell Alloimmunization After Intrauterine TransfusionsSanquin-LUMC J.J van Rood Center for Clinical Transfusion ResearchHemolytic disease of the fetus and newborn, HDFN, Red blood cell alloimmunizationApr 2017Aug 2020
Evaluation of Knowledge Among Adolescents With Sickle Cell Disease.Adele CARLIER-GONODMar 2017Mar 2018
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell DiseaseNational Heart, Lung, and Blood Institute (NHLBI)Peripheral Blood Stem Cells, Host-Donor Chimerism, Graft Versus Host Disease, Haploidentical, Donor ApheresisMar 2017Aug 2026
Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label TrialAla'a ShararaThalassemia, HCV, Sofosbuvir, LedipasvirFeb 2017Aug 2017
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)University of California, San Franciscointrauterine transfusion, in utero human stem cell transplantationFeb 2017Feb 2024
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage StrategiesSt. Jude Children's Research HospitalSickle cell, Hydroxyurea, InfantsFeb 2017Jun 2020
New Hemolysis Parameters in Sickle Cell DiseaseQueen Fabiola Children's University HospitalFeb 2017Feb 2019
Pathophysiology of Acute Pain in Patients With Sickle Cell DiseaseNational Heart, Lung, and Blood Institute (NHLBI)Pain, Cell Free DNA, Inflammatory Markers, Microparticles, NETosisJan 2017Oct 2021
Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain ManagementKlein Buendel, Inc.Sickle Cell Disease, Adolescence, Pain Assessment, Tablet Application, Pain, mHealthJan 2017Sep 2017
Omega-3 Fatty Acids in Sickle Cell DiseaseRobin E. MillerSickle Cell Disease, omega-3 fatty acids, Docosahexaenoic Acid (DHA), Eicosapentaenoic Acid (EPA)Jan 2017Apr 2019
Dose-Finding Study of SC411 in Children With Sickle Cell DiseaseSancilio and Company, Inc.Jan 2017Jul 2017
A Pilot Study of Fecal Microbiome and Neutrophil Cellular Adhesion Molecules in Patients With Sickle Cell Disease (SCD)Rhode Island HospitalJan 2017Dec 2017
A Phase Ib Study of NVX-508 in Sickle Cell DiseaseAmma Owusu-Ansah, MDJan 2017Dec 2017
A Study of EPEG in Beta Thalassemia PatientsProlong PharmaceuticalsDec 2016Jun 2017
Precision Medical Research of Non-immune Fetal Hydrops (NIFH)-From Prenatal Diagnosis to Intrauterine TreatmentShanghai First Maternity and Infant HospitalDec 2016Aug 2018
The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia MajorCairo UniversityBeta Thalassemia, Metformin, Oxidative stress, Iron overload, Liver functionDec 2016May 2018
Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell DiseaseAlessandra BiffiPlerixaforDec 2016Jan 2018
Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)Global Blood TherapeuticsDec 2016Jun 2019
Fetal Hemoglobin Induction Treatment MetforminBaylor College of MedicineMetformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease; Sickle-Cell, Thalassemia, Hemoglobin DiseaseNov 2016Nov 2022
A Study of IMR-687 in Healthy Adult VolunteersImara, Inc.Sickle Cell Disease, Sickle Beta 0 ThalassemiaNov 2016Jun 2017
SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell PainChildren's Research InstituteNov 2016Sep 2018
Hydoxyurea Exposure in Lactation: A Pharmacokinetics Study (HELPS)Children's Hospital Medical Center, CincinnatiHydroxyureaOct 2016Nov 2019
Monocytic Expression of Heme Oxidase-1 (HO-1) in Sickle Cell Patients and Correlation With the Humoral Immune Response to Vaccine and With Allo-immunization.Francis CorazzaSickle Cell Disease, Heme oxidase-1 (HO-1)Oct 2016Oct 2018
Oxygen Therapy and Pregnancy in Sickle Cell DiseaseAssistance Publique - Hôpitaux de Parissickle cell disease, oxygen therapy, pregnancy, pregnancy in sickle cell diseaseSep 2016Jun 2021
Mobile-Directly Observed Therapy on Adherence to HydroxyureaMuhimbili University of Health and Allied SciencesSickle cell disease, Hydroxyurea, Adherence, randomized trial, medication possession ratioSep 2016Aug 2017
Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell DiseaseVanderbilt Universitysickle cell disease, pulmonary complications, reduced FEV1, azithromycin, lung functionSep 2016Oct 2018
The Effective and Safety of Thalidomide in NTDTXiao-Lin YinThalidomide NTDTSep 2016Mar 2018
Patient-Empowered Mobile Technology in Hospitalized Patients: Technology Resources to Understand Pain Assessment in Patients With Pain (TRU-PAIN)Duke Universityoncology, bone marrow transplant, sickle cell diseaseAug 2016Aug 2018
Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of ChimerismFirst Affiliated Hospital of Guangxi Medical Universitythalassemia, transplantation, chimerism, donor ymphocytesAug 2016Dec 2017
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) FormulationNovartis PharmaceuticalsAnemia., Myelodysplastic Syndrome, Thalassemia, Film coated tabletJul 2016Sep 2018
Ketamine as an Adjuvant Therapy for Acute Vaso Occlusive Crisis in Pediatric Patients With Sickle Cell DiseaseGeorgia Regents UniversityJul 2016Jul 2018
Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell DiseaseMonica BhatiaSickle Cell Disease, Graft Versus Host Disease, Abatacept, Hematopoietic Stem Cell TransplantationJul 2016Dec 2017
Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)Medical College of WisconsinSickle Cell Disease, Young Adults, Phase II Trial, Hematopoietic Cell Transplantation (HCT), Human Leukocyte Antigen (HLA)Jun 2016Oct 2022
Benefits of Nigella Sativa in Children With Beta Thalassemia MajorTanta UniversityJun 2016Aug 2016
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive CrisisChildren's Hospital & Research Center OaklandKetamine, Sickle Cell Disease, Vaso-Occlusive CrisisJun 2016Dec 2018
Zinc Supplementation in Children With Sickle Cell Disease in Western KenyaLucas Otieno Tina, MD MScChildren, Sickle Cell Disease, ZincMay 2016Jan 2017
Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent TransfusionsDuke UniversityMay 2016Jun 2017
SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell AnemiaChildren's Hospital Medical Center, CincinnatiMay 2016Oct 2020
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning RegimenNational Guard Health AffairsMay 2016Dec 2019
Identifying Barriers and Strategies to Support Self-efficacy for Medication Adherence With Text MessagingVanderbilt UniversityMay 2016May 2017
Study of Hydroxyurea to Treat Sickle Cell DiseaseMersin UniversitySickle cell disease, Hydroxyurea, ComplianceMay 2016Jul 2016
A Multi-Center Study of Riociguat in Patients With Sickle Cell DiseasesGregory J. Kato, MDSCD, Sickle Cell Disease, Riociguat, AdempasApr 2016Jun 2019
A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease PatientsProlong PharmaceuticalsApr 2016Sep 2017
The Feasibility of the PAINReportIt Guided Relaxation Intervention-INPATIENTUniversity of FloridaApr 2016Apr 2018
Transplantation Using Reduced Intensity Approach for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone MarrowEmory Universitybone marrow transplantApr 2016Dec 2020
Improving Self-Management in Adolescents With Sickle Cell DiseaseChildren's Hospital Medical Center, CincinnatiAnemia, Sickle Cell, Self Care, Adolescent, Young Adult, Disease Management, Behavioral Activation, Peer CommunicationApr 2016Apr 2018
Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell DiseaseWashington University School of MedicineMar 2016May 2020
Investigation of the Genetics of Hematologic DiseasesSt. Jude Children's Research HospitalGenetics, Whole genome sequencing, Pediatrics and hematologyMar 2016Aug 2026
Plasma DNA and Vascular Remodelling in Patients With Sickle Cell DiseaseADDMEDICA SASAMar 2016Sep 2018
Evaluating Thromboelastography (TEG) and ETP in Sickle AdultsGuy's and St Thomas' NHS Foundation TrustMar 2016Jun 2016
Myeloablative Conditioning and Haplo AlloSCT for Patients With Sickle Cell DiseaseNew York Medical Collegestem cell transplantation, sickle cell disease, haploidentical, defibrotideFeb 2016Dec 2021
Screening for Alpha Thalassemia in Healthy VolunteersNational Heart, Lung, and Blood Institute (NHLBI)Alpha Thalassemia, Double DeletionFeb 2016Dec 2018
A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest SyndromeUniversity of Mississippi Medical Centersickle cell disease, citrullineFeb 2016Dec 2017
Arginine Therapy for Sickle Cell Disease PainEmory UniversityArginine TherapyJan 2016Aug 2019
SANGUINATE™ in Sickle Cell Disease Associated Leg UlcerProlong PharmaceuticalsLeg Ulcer, LU, SANGUINATE, QOL, SCD, Sickle Cell Disease, Sickle Cell, Anemia, Leg Wound, Leg, Skin LesionJan 2016Jan 2017
Multi-center Study of SC411 for Sickle Cell DiseaseSancilio and Company, Inc.hemoglobin SS, hemoglobin SC, hemoglobin S/β°-thalassemiaJan 2016Dec 2017
Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell DiseaseMontefiore Medical CenterJan 2016Jun 2018
Measures of Respiratory Health (MRH)The Hospital for Sick ChildrenCF, Multiple Breath Washout, Lung Clearance Index, PediatricsJan 2016Dec 2018
Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)Baylor College of MedicineDec 2015Dec 2020
Effect of Spirulina on Serum Hyaluronic Acid in Beta Thalassemic Children With Hepatitis CTanta UniversityDec 2015Dec 2016
Effect of Spirulina on Liver Fibrosis by Transient Elastography in Beta Thalassemic Children With Hepatitis CTanta UniversityDec 2015Dec 2016
The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj HospitalMahidol UniversityTransient elastography, FibrotestNov 2015Feb 2017
Sub-dissociative Intranasal Ketamine for Pediatric Sickle Cell Pain CrisesCameroon Baptist Convention HealthKetamine, Intranasal, Pain crisis, Vasoocclusive Pain, Sickle cell diseaseOct 2015Feb 2017
the Potential Immunomodulatory Effects of Spirulina on Thalassemic ChildrenTanta UniversityOct 2015Oct 2017
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)ADDMEDICA SASAPharmacokinetics of HydroxyureaSep 2015Jun 2016
Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid SupplementsSultan Qaboos UniversityPrevention of Vaso Occlusive Painful CrisisSep 2015Mar 2019
Evaluation of the Hemostatic Potential in Sickle Cell Disease PatientsBrugmann University HospitalHemostatic potential, Sickle cell disease, Thrombin generation testSep 2015Sep 2017
A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy SubjectsGlobal Blood TherapeuticsSep 2015Feb 2016
Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy SubjectsGlobal Blood Therapeuticsanemia, sickle cellSep 2015Feb 2016
Comparison of Two Methods of Transfusion for Stroke Prevention in Sickle CellChattanooga-Hamilton County Hospital AuthorityAug 2015Jun 2017
Comparison of Patient Centered Outcomes for People With SCD in the Acute Care SettingJohns Hopkins UniversityJul 2015Dec 2018
Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell DiseasePfizerRivipansel, GMI-1070, Selectin Inhibitor, SCD, VOCJul 2015Feb 2020
Sevuparin Infusion for the Management of Acute VOC in Subjects With SCDDilaforette ABJul 2015Jun 2016
Carbon Monoxide Measurement to Screen for Sickle Cell DiseaseChildren's Hospital & Research Center OaklandSickle Cell AnemiaJul 2015Dec 2015
Macitentan in Pulmonary Hypertension of Sickle Cell DiseaseBoston UniversityPulmonary Hypertension, Sickle Cell Disease, Pulmonary Arterial Hypertension, MacitentanJul 2015Dec 2016
Evaluation of Repeat Administration of Purified Poloxamer 188Mast Therapeutics, Inc.Sickle cell, vaso-occlusive crisisJun 2015Jun 2016
Music Therapy in Sickle Cell Transition StudyUniversity Hospital Case Medical CenterJun 2015Aug 2016
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia PatientsKevin H.M. Kuo, MD, MSc, FRCPCJun 2015Jun 2018
SMART Mobile Application Technology Utilization in the Treatment of Sickle Cell Disease Post Day Hospital DischargeDuke UniversitySickle Cell DiseaseJun 2015May 2017
A Study to Evaluate the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell DiseaseAstraZenecaSickle cell disease, Young adults, Hestia2, TicagrelorJun 2015Jun 2016
A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male SubjectsGlobal Blood TherapeuticsJun 2015Dec 2015
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other AnemiasApoPharmaIron overload, Sickle cell disease, Deferiprone, Ferriprox, Iron chelationMay 2015Oct 2018
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell DiseaseEmory UniversityMay 2015Jul 2019
Gene Therapy for Transfusion Dependent Beta-thalassemiaIRCCS San RaffaeleBeta thalassemia, Gene therapy, Lentiviral vectorMay 2015Aug 2019
Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive CrisisProlong PharmaceuticalsApr 2015Apr 2016
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta ThalassemiaTanta UniversityApr 2015Apr 2017
Mental Health Treatment in Sickle Cell DiseaseUniversity of PittsburghMar 2015Dec 2015
Effect of Spirulina on Liver Iron Concentration in Beta Thalassemic Children With Hepatitis CTanta UniversityMar 2015Dec 2016
Comparing Acute Pain Management Protocols for Patients With Sickle Cell DiseaseDuke UniversitySickle Cell Disease, Emergency Department, Vaso-occlusive Crisis, Pain Management, Pilot ProjectFeb 2015Sep 2016
Dose-Escalation Study of SCD-101 in Sickle Cell DiseaseInvenux, LLCHomozygous Sickle Cell Disease S/Beta 0 ThalassemiaFeb 2015Jan 2016
Sperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants?Ain Shams UniversityFeb 2015Dec 2017
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell DiseaseGlobal Blood TherapeuticsJan 2015May 2016
Ledipasvir/Sofosbuvir Fixed-Dose Combination for 12 Weeks in Genotype 1 or 4 HCV Infected Adults With Sickle Cell DiseaseGilead SciencesSickle Cell Disease, Gilead, Hepatitis CDec 2014Mar 2016
Treatment of Hemoglobin Sickle Cell DiseaseBaylor College of MedicineHemoglobin SC disease, hydroxyurea, quality of life, viscosity, red cell densityDec 2014Dec 2019
PATient Navigator to rEduce ReadmissionsUniversity of Illinois at ChicagoPatient Navigators, Readmissions, Peer CoachingOct 2014Apr 2016
A New Reagent Assay Examining Natural Parvovirus B19 Infection in Sickle Cell DiseaseSt. Jude Children's Research HospitalParvovirus B19 InfectionOct 2014May 2017
ACE-536 Extension Study - Beta ThalassemiaAcceleron Pharma, Inc.Oct 2014Apr 2017
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-ThalassemiaSideris PharmaceuticalsChelation, Iron Overload, Thalassemia, Transfusion, Iron ChelationOct 2014Jul 2015
Feasibility and Efficacy of a Home-based, Computerized Cognitive Training Program in Pediatric Sickle Cell DiseaseSteven J. HardyNeurocognitive, Working memory, Cogmed, Cognitive trainingOct 2014Aug 2017
Vitamin D3 in Patients With Sickle Cell DiseaseIcahn School of Medicine at Mount SinaiSickle Cell Disease, Vitamin DOct 2014Dec 2017
Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell PatientsAssistance Publique - Hôpitaux de ParisSickle cell disease, Open monocenter study, Phase I/II, hematopoietic stem cells, PlerixaforSep 2014Jun 2017
A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor Followed by a 4 Weeks Extension Phase in Paediatric Patients With Sickle Cell DiseaseAstraZenecasickle cell anemia, paediatricSep 2014Sep 2015
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesBellicum PharmaceuticalsSevere Combined Immune Deficiency, Congenital T-cell Defect, Congenital T-cell Deficiency, Chronic Granulomatous Disease, Shwachman Diamond Syndrome, Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Sickle Cell Disease, Thalassemia, Mucopolysaccharidosis, SphingolipidosesSep 2014Dec 2018
Music Therapy in Sickle Cell Pain RCTUniversity Hospital Case Medical CenterMusic Therapy, Sickle Cell Disease, PainSep 2014Jun 2016
Decision Aid for Therapeutic Options In Sickle Cell DiseaseEmory UniversityHydroxyurea, Bone marrow transplant, Chronic blood transfusion, Treatment options for sickle cell disease, Decision makingSep 2014Dec 2016
Denosumab vs Placebo in Patients With Thalassemia Major and OsteoporosisErsi VoskaridouSep 2014Dec 2016
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Diseasebluebird bioAug 2014Jan 2018
Haploidentical Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease and Thalassemia Using CD34+ Positive Selected GraftsCatherine BollardAug 2014Dec 2019
Fertility Preservation in Women Who Will be Undergoing Gonadotoxic Therapy, Hematopoietic Stem Cell Transplantation, and in Women With Sickle Cell DiseaseEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Bone Marrow Transplant, Fertility, OvaryAug 2014Aug 2019
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell DiseaseNational Heart, Lung, and Blood Institute (NHLBI)Hydroxyurea, Sickle Cell Disease, Fetal Hemoglobin InductionAug 2014May 2018
Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell DiseaseSt. Jude Children's Research HospitalAug 2014Mar 2017
Study of Beet Juice for Patients With Sickle Cell AnemiaDaniel Kim-ShapiroErythrocyte Deformability, Platelet Activation, Platelet Aggregation, Nitrite, Sickle Cell Anemia, Diet TherapyJul 2014Apr 2015
Stem Cell Gene Therapy for Sickle Cell DiseaseDonald B. Kohn, M.D.Sickle Cell Disease (SCD), Gene Therapy, Lentiviral Vector, Beta GlobinJul 2014Apr 2018
Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell DiseasePfizerJun 2014Apr 2015
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia MajorAffiliated hospital of guangxi medical university,chinabeta-Thalassemia majorJun 2014Dec 2018
Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell DiseaseEmory UniversityJun 2014Dec 2018
Sickle Cell Disease (SCD) Decision AidEmory UniversityMay 2014Mar 2015
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)Novartis PharmaceuticalsIron overload, Chelation, Thalassemia, Myelodysplastic syndrome (MDS), Transfusional hemisiderosis, Deferasirox, ICL670, Dispersible tablet, Film-coated tabletMay 2014Dec 2015
Use of Mobile Technology for Intensive Training in Medication ManagementDuke UniversitySickle cell, Thalassemia, AdherenceMay 2014May 2016
Medication Adherence in Youth With Sickle Cell Disease (SCD)St. Jude Children's Research HospitalMedication Adherence, HydroxyureaMay 2014May 2015
Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg UlcersNational Human Genome Research Institute (NHGRI)Genetic Disorders, Sickle Cell Disease, African American, Leg Ulcers, SkinMay 2014Apr 2016
Sickle Cell Hemoglobinopathies and Bone HealthUniversity of Connecticut Health CenterSickle Cell Disease (SCD), Sickle Cell Trait (SCT)May 2014May 2017
Feasibility Study of Unfractionated Heparin in Acute Chest SyndromeUniversity of PittsburghSickle cell disease, Acute chest syndrome, Hemoglobinopathy, Hemolytic anemia, Heparin, AnticoagulantApr 2014Apr 2016
Gum Arabic as Fetal Hemoglobin Agent in Sickle Cell AnemiaAl-Neelain UniversityApr 2014Jan 2015
Androgen Regulation of Priapism in Sickle Cell DiseaseJohns Hopkins UniversityPriapism, Sickle cell disease, MaleMar 2014Sep 2017
Hematopoietic Stem Cell Transplant for Sickle Cell DiseaseCase Comprehensive Cancer CenterSickle Cell Disease, Sickle Cell Anemia, GvHD, fludarabineMar 2014Nov 2016
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent ThalassemiaNovartis PharmaceuticalsEndocrine complications, transfusion dependent thalassemiaMar 2014Mar 2018
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant FailureNational Heart, Lung, and Blood Institute (NHLBI)Sickle Cell Disease, Allogeneic Hematopoietic Stem Cell Transplant, Pentostatin (Nipent), Cyclophosphamide, Alemtuzumab (Campath)Mar 2014Aug 2021
Sickle Cell Clinical Research and Intervention ProgramSt. Jude Children's Research HospitalSickle Cell Anemia, Sickle Cell Disease, Survival, Mortality, End-Order DysfunctionMar 2014Dec 2044
Hepcidin Levels in Sickle Cell Disease (SCD)Kenneth Ataga, MDsickle cell diseaseMar 2014May 2015
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other AnemiasApoPharmasickle cell disease, Iron overload, Deferiprone, Ferriprox, deferoxamine, ChelationFeb 2014Mar 2016
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.Novartis Pharmaceuticalsthalassemia, spleen enlargementFeb 2014Feb 2016
Amlodipine for Myocardial Iron in ThalassemiaAga Khan UniversityThalassemia, Amlodipine, Chelation, T2*, MRI, Myocardial IronFeb 2014May 2015
The Effect of Rivaroxaban in Sickle Cell DiseaseUniversity of North Carolina, Chapel Hillsickle cell anemia, sickle cell disease, rivaroxaban, direct Xa inhibition, coagulation, anticoagulationFeb 2014Sep 2016
Novel Use Of Hydroxyurea in an African Region With MalariaUniversity of Minnesota - Clinical and Translational Science InstituteHydroxyureaJan 2014Jan 2017
Allograft for Sickle Cell Disease and ThalassemiaUniversity of Texas Southwestern Medical CenterSickle Cell Disease, ThalassemiaJan 2014Jan 2018
Reduced Intensity Conditioning in Patients Aged ≤30 With Non-Malignant Disorders Undergoing Cord Blood TransplantationUniversity of PittsburghSevere Combined Immune Deficiency (SCID) with NK cell activity, Omenn Syndrome, Bare Lymphocyte Syndrome (BLS), Combined Immune Deficiency (CID) syndromes, Combined Variable Immune Deficiency (CVID) syndrome, Wiskott-Aldrich Syndrome, Leukocyte adhesion deficiency, Chronic granulomatous disease (CGD), X-linked Hyper IgM (XHIM) syndrome, IPEX syndrome, Chediak -Higashi Syndrome, Autoimmune Lymphoproliferative Syndrome (ALPS), Hemophagocytic Lymphohistiocytosis (HLH) syndromes, Lymphocyte Signaling defects, Other primary immune defects where HSCT may be beneficial, Dyskeratosis Congenita (DC), Congenital Amegakaryocytic Thrombocytopenia (CAMT), Osteopetrosis, Mucopolysaccharidoses, Hurler syndrome (MPS I), Hunter syndrome (MPS II), Sanfilippo syndrome (MPS II), Leukodystrophies, Krabbe Disease, also known as globoid cell leukodystrophy, Metachromatic leukodystrophy (MLD), X-linked adrenoleukodystrophy (ALD), Other inherited metabolic disorders, alpha mannosidosis, Other inheritable metabolic diseases where HSCT may be beneficial., Thalassemia major, Sickle cell disease (SCD), Diamond Blackfan Anemia (DBA), other congenital transfusion dependent anemiasDec 2013Nov 2018
Survey in a Population of Sickle Cell Disease Patients to Evaluate the Transition Between the Queen Fabiola Children Hospital and the CHU Brugmann Hospital, and the Quality of the Hospital Care Within the CHU Brugmann Hospital.Brugmann University Hospitalpediatric hospital care, adult hospital care, sickle cell diseaseDec 2013Dec 2016
Phase 2 Study of Montelukast for the Treatment of Sickle Cell AnemiaVanderbilt UniversityNov 2013Oct 2017
SCD-Haplo: A Phase II Study of HLA-Haploidentical Stem Cell Transplation to Treat Clinically Aggressive Sickle Cell DiseaseDamiano Rondelli, MDNov 2013Oct 2018
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell TransplantDiane George, MDUnrelated donor transplant, Allogenic Stem Cell Transplant, Adult Bone Marrow Transplant, Pediatric Bone Marrow Transplant, Related donor transplant, Haploidentical donor transplant, Peripheral blood stem cell transplantation, Non-malignant disease, Malignant disease, Bone marrow failure syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndrome, Acquired Immunodeficiency Syndrome, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Reduced-Intensity Conditioning, Sickle Cell Disease, Sickle Cell-beta-thalassemiaNov 2013Dec 2017
Added Value of Speckle Tracking in the Evaluation of Patients With Sickle Cell DiseaseBrugmann University HospitalSickle Cell Disease, Echocardiography, Speckle trackingNov 2013Mar 2016
Feasibility of a Stress Reduction Intervention Study in Sickle Cell DiseaseUniversity of Illinois at ChicagoNov 2013Nov 2014
Ph. 2 Study of MP4CO to Treat Vaso-occlusive Sickle CrisisSangartSickle cell anemia, Sickle cell disease, Sickling crisis, Vaso-occlusive crisis, Carboxyhemoglobin, Oxygen therapeutic, Ischemic rescue therapy, Hemoglobin solution, Pegylated hemoglobinOct 2013Oct 2015
Treatment of Iron Overload Requiring Chelation TherapyShire Development LLCOct 2013Jul 2015
Pain Management in Children and Young Adults With Sickle Cell DiseaseSt. Jude Children's Research HospitalPain controlOct 2013Oct 2017
Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell DiseaseSt. Jude Children's Research HospitalNocturnal enuresis, Barriers to treatmentOct 2013Oct 2015
Hydroxyurea Therapy, Sickle Cell Disease, NDEPTHBaylor College of MedicineSickle cell, Hydroxyurea, Dose prediction, Maximum tolerated doseOct 2013Oct 2018
Utility of Fibroscan in Estimating Hepatic Iron ConcentrationUniversity of British ColumbiaBeta Thalassemia MajorOct 2013Oct 2014
Abciximab (ReoPro) as a Therapeutic Intervention for Sickle Cell Vaso-Occlusive Pain CrisisSt. Louis UniversitySickle Cell Pain Crisis, Abciximab, Integrins, Cell Adhesion MoleculesSep 2013Sep 2015
Deferasirox BID in Transfusion Dependent Thalassemia Patients With Indadequate Response to High DosesNovartis PharmaceuticalsSep 2013Sep 2015
Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment: HABITColumbia UniversitySickle cell disease, Medication adherence, Hydroxyurea, Community health worker intervention, Sickle Cell TreatmentSep 2013May 2015
Pneumococcal Vaccination for Splenectomised Thalassemia Major Patients in IndonesiaFakultas Kedokteran Universitas IndonesiaSep 2013Feb 2014
Quantifying the Presence of Lung Disease and Pulmonary Hypertension in Children With Sickle Cell DiseaseDuke Universitysickle cell disease, pulmonary hypertension, airway hyperreactivity, acute chest syndromeAug 2013Feb 2020
Improving Disease Knowledge in Adolescents With Sickle Cell DiseaseThe University of The West IndiesEducational intervention, Formal Counselling, Knowledge, Illness PerceptionAug 2013Aug 2015
Evaluation of Sickle Cell Liver DiseaseNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Microbiome, Sickle Cell Disease, Bone Marrow TransplantAug 2013May 2018
Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain CrisesSelexys Pharmaceuticals CorporationSelG1, P-selectin, monoclonal antibody, sickle cell disease, sickle cell anemia, sickle cell, pain crisis, pain crises, vasoocclusion, vaso-occlusion, priapism, hepatic sequestration, splenic sequestration, chest syndromeJul 2013Aug 2015
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years OldNovartis PharmaceuticalsdeferasiroxJun 2013Oct 2014
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric PatientsConsorzio per Valutazioni Biologiche e Farmacologichechronic iron overload, hereditary haemoglobinopathy, beta thalassaemia major, chelating agents, deferiprone, deferasirox, children, paediatricsJun 2013Dec 2014
Exhaled Carbon Monoxide as a Marker of Hemolysis in Sickle Cell Disease- an Exploratory StudyUniversity Hospital Case Medical Centercarbon mono oxide, sickle cell, hemolysisJun 2013Jun 2015
Nitrous Oxide Analgesia Vaso-occlusive CrisisColumbia UniversitySickle cell disease, Vaso-occlusive crisis, Nitrous oxide, Emergency departmentJun 2013Jun 2014
Efficacy, Safety Study and Benefit of Alkali Therapy in Sickle Cell DiseaseUniversity Hospital Case Medical CenterSickle cell disease, Chronic kidney disease, Bicarbonate therapyJun 2013Jun 2015
Reproductive Capacity and Iron Burden in ThalassemiaChildren's Hospital & Research Center OaklandInfertility, Thalassemia, Iron overload, Pituitary Iron Load, Oxidative Stress, REPRODUCTIVE CAPACITY AND ASSOCIATION TO IRON BURDEN AND CHELATION PATTERNS IN THALASSEMIA MAJOR PATIENTSJun 2013Jun 2016
Evaluation of the Free α-hemoglobin Pool in the Red Blood Cells : Prognostic Marker and Severity Index in Thalassemic SyndromesAssistance Publique - Hôpitaux de ParisAlpha-Hemoglobin Stabilizing Protein, alpha-Hb pool, thalassemias, diagnostic method, biomarkerJun 2013Jun 2016
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell DiseaseApoPharmasickle cell disease, iron overload, pharmacokinetics, Ferriprox, deferiproneMay 2013Jul 2013
Non-Myeloablative Conditioning and Bone Marrow TransplantationVanderbilt-Ingram Cancer CenterMay 2013May 2016
A Phase II Trial of Regadenoson in Sickle Cell AnemiaDana-Farber Cancer InstituteApr 2013Apr 2015
N-Acetylcysteine in Patients With Sickle Cell DiseaseAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)Sickle Cell Disease, Sickle Cell Anemia, Pain, N-Acetylcysteine, Acetylcysteine, Oxidative stressApr 2013Oct 2014
A Study Evaluating the Safety and Efficacy of the Gene Therapy LentiGlobin® BB305 Drug Product in Subjects With Beta-Thalassemia MajorBluebird Biogene therapy, beta thalassemia, hemoglobin, anemia, CD34Mar 2013Jul 2017
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)Eli Lilly and CompanyMar 2013Jun 2015
CD34+ Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation for Non-Malignant DiseaseDiane George, MDUnrelated donor transplant, Haploidentical donor transplant, Peripheral blood stem cell transplantation, CD34+ selection, Non-malignant disease, Bone marrow failure syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndrome, Acquired Immunodeficiency Syndrome, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemiaMar 2013Mar 2017
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)NKT TherapeuticsSickle cell diseaseFeb 2013Aug 2014
Sickle Cell Disease - Stroke Prevention in Nigeria TrialVanderbilt Universitytranscranial Doppler, stroke, sickle cell anemia, Nigeria, hydroxyurea, low income countryFeb 2013Aug 2014
Microvascular and Fibrosis Imaging StudyDuke UniversityAutoimmune diseases with cutaneous involvement, Systemic sclerosis, Scleroderma, Morphea, Dermatomyositis, Cutaneous lupus, Vasculitis, Sickle Cell Disease, Graft versus Host DiseaseJan 2013Jan 2017
Vaporized Cannabis for Chronic Pain Associated With Sickle Cell DiseaseUniversity of California, San FranciscoJan 2013Mar 2016
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC TransfusionCerus CorporationS303 treated RBCsDec 2012Jun 2014
Pharmacokinetic Study of Deferiprone in Paediatric PatientsConsorzio per Valutazioni Biologiche e Farmacologichechronic iron overload, hereditary haemoglobinopathy, beta thalassaemia majorDec 2012Apr 2013
Evaluation of Purified Poloxamer 188 in Children in Crisis (EPIC)Adventrx Pharmaceuticalssickle cell disease, vaso-occlusive crisisDec 2012Aug 2015
An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)Novartis PharmaceuticalsDec 2012Jul 2014
Study of ACE-536 to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia IntermediaAcceleron Pharma, Inc.Dec 2012Nov 2014
Evaluation of Spectra Optia RBC Exchange in Sickle Cell PatientsCaridianBCTNov 2012May 2013
Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood DisordersMassachusetts General HospitalKidney, Chronic Kidney Disease, CKD, Bone Marrow, Bone Marrow Transplant, BMT, Leukemia, AML, ALL, CML, CLL, MM, NHL, MDSNov 2012Jan 2015
Zinc and Diabetes in Patients With Thalassemia: a Pilot StudyChildren's Hospital & Research Center OaklandThalassemia, Diabetes, Zinc, Iron-OverloadNov 2012Dec 2014
The Use of Warmed Saline in Vaso-occlusive EpisodesNationwide Children's HospitalNov 2012Jun 2014
Single-center Prospective Evaluation of Sickle Cell Patient Care in the CHU Brugmann Emergency DepartmentBrugmann University HospitalSickle Cell Disease, Vaso-occlusive crisis, Emergency managementNov 2012May 2015
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent ThalassemiaNovartis PharmaceuticalsNon-transfusion dependent thalassemia, NTDTOct 2012Apr 2014
Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II ProtocolNational Heart, Lung, and Blood Institute (NHLBI)Oct 2012Dec 2016
Cognitive Rehabilitation in Sickle Cell DiseaseDuke Universitysickle cell disease, pediatric, learning disability, cognitive impairment, memory, attention, Children with sickle cell diseaseOct 2012Mar 2014
Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without HemolysisAshutosh LalOct 2012Oct 2013
Sickle Cell Trait in Football PlayersUniversity of South FloridaSickle Cell Trait, Football, Sickling, Heat Illness, SCTOct 2012Aug 2013
Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient NavigatorsVirginia Commonwealth Universitysickle cell disease, hydroxyurea, patient navigator, community health worker, public health worker, fetal hemoglobin, health services researchOct 2012Aug 2016
Combination Deferasirox and Deferiprone for Severe Iron Overload in ThalassemiaChildren's Hospital of PhiladelphiaThalassemia, Chelation, Transfusion, SafetySep 2012Aug 2015
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia MajorNanfang Hospital of Southern Medical UniversityCord Blood Transplant, Haploidentical HSCT, Thalassemia majorSep 2012Jun 2015
ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin GeneMemorial Sloan-Kettering Cancer CenterBUSULFAN, G-CSF, CliniMACS-CD34 Reagent System, Blood transfusion, 10-164, Autologous CD34+ cells transduced with TNS9.3.55, a lentiviral vector encoding the human β-globin geneJul 2012Jul 2014
Liver Fibrosis in Sickle Cell DiseaseUniversity of Miamisickle cell disease, hemochromatosis, liver fibrosis, liver cirrhosis, FibroScan, MRI R2*, liver ironJul 2012Dec 2017
Post Hematopoietic Stem Cell TransplantationNovartis Pharmaceuticalshematopoietic stem cell transplantation, beta-thalassemia major, deferasirox, iron overloadJun 2012Jul 2014
Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.Ain Shams UniversityHydroxyurea, Erythropoitin therapy, Thalassemia intermediaJun 2012Dec 2012
Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia MajorShiraz University of Medical SciencesFocus, immunomodulatory effect, of silymarin, on cell mediated immunity, Beta-Thalassemia, major patients.Jun 2012Sep 2012
Use of a Mobile-based App for SCD PatientsJude C. JonassaintSickle cell, PainJun 2012Jun 2014
Trial of HQK-1001 in Beta Thalassemia Intermedia in LebanonBoston UniversityThalassemia intermedia, Fetal hemoglobin, HemoglobinMay 2012Jan 2013
Massive Iron Deposit AssessmentSt. Jude Children's Research HospitalSickle cell disease, Thalassemia, Hemochromatosis, CancerApr 2012Apr 2017
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.Celgene CorporationBeta-ThalassaemiaMar 2012Jun 2014
Study of SDMB in Beta Thalassemia Intermedia in ThailandBoston UniversityThalassemia, Anemia, Fetal hemoglobinMar 2012Dec 2012
Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused PopulationsAssistance Publique - Hôpitaux de ParisThalassemia, sickle cell disease, myelodysplasia, transfusion, chelationMar 2012Mar 2015
Effects of Transfusion of Older Red Blood Cells on Patients With HemoglobinopathiesColumbia Universityiron, transfusion, red blood cells, sickle cell disease, thalassemia, blood donorsFeb 2012Feb 2015
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron OverloadAin Shams UniversityIron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasirox, Deferiprone, 1- Beta-thalassemia major patients;, Patients with high iron stores, Serum ferritin consistently > 2500 mcg/l and or increasing trend over previous 12 months, Liver iron >14 mg/g dry weight- by R2 MRI, 2- Sickle cell disease, 3- Other causes of transfusional iron hemosiderosisFeb 2012Feb 2013
Prevalence of Pulmonary Hypertension (PAH) in Patients With ThalassemiaEnte Ospedaliero Ospedali GallieraThalassemia, Pulmonary Arterial HypertensionJan 2012Jan 2013
Safety Study of MP4CO in Adult Sickle Cell PatientsSangartSickle cell anemia, Sickle cell disease, Sickling crisis, Vaso-occlusive crisis, Carboxyhemoglobin, Oxygen therapeutic, Hemoglobin solutions, Pegylated hemoglobinJan 2012Mar 2013
Effect of Metoprolol on Thalassemia CardiomyopathyShiraz University of Medical SciencesThalassemia cardiomyopathy, metoprolol succinate, echocardiography, six minute walk testJan 2012Dec 2012
Functional Neuroimaging of Pain Using EEG and fMRIUniversity of Minnesota - Clinical and Translational Science InstituteSickle CellJan 2012May 2015
Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia IntermediaHaEmek Medical Center, IsraelBeta globin gene, Alfa globin gene, xmn1 polymorphismOct 2011Dec 2012
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)Center for International Blood and Marrow Transplant ResearchOct 2011
Providing Access to Cord Blood Units for TransplantsNational Cancer Institute (NCI)Adult and Pediatric, Unlicensed Cord Blood Unit IND, NMDP, Transplant, Hematologic Malignancies, Leukemia, Multiple Myeloma, Non-Hodgkin Lymphoma, CML, Chronic Myelogenous Leukemia, Myelodyplastic Syndrome, MDS, Thalassemia, Lysosomal Storage DiseasesOct 2011Nov 2012
Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia MajorUniversity of Campinas, BrazilMagnetic resonance imaging, Thalassemia, Iron overloadAug 2011Dec 2013
Study of Effects of YisuiShengxueGranules on ThalassemiaGuang'anmen Hospital of China Academy of Chinese Medical Sciencestraditional chinese medicine and thalassemiaJul 2011Jan 2012
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron OverloadShire Development LLCBeta-Thalassemia, Sickle Cell Anemia, Transfusional iron overload, Iron Overload, Iron ChelationJul 2011Dec 2013
The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response StudyWeill Medical College of Cornell UniversityThalassemia, hypercalciuria, vitamin D, To conduct a pilot study to determine the effect of various doses of, vitamin D supplementation on vitamin D stores and their association with calcium excretionJun 2011Dec 2014
Stem Cell Transplantation for Sickle Cell AnemiaHackensack University Medical CenterStem cell transplant, Sickle cell, Stem cell transplantation, Related, UnrelatedJun 2011Jun 2018
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell DiseaseChildren's Hospital Medical Center, CincinnatiAnemia, Sickle Cell, Transition to Adult Care, Self Care, Personal Electronic Health Records, Adolescent, Young Adult, Disease Management, Physician-Patient RelationsJun 2011Jul 2015
Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesUniversity of Louisvilleother hemoglobinopathies, chimerism, sickle cell, thalassemia, Marrow/Enriched Hematopoetic Stem Cell TransplantMay 2011May 2030
High-Tc Susceptometer to Monitor Transfusional Iron OverloadColumbia UniversityIron, Iron overload, Hepatic iron concentration, High-temperature superconductivity, Magnetic susceptibility, SusceptometerMar 2011May 2014
Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood DisordersNational Heart, Lung, and Blood Institute (NHLBI)Sickle Cell Disease, Hemolysis, Anemia, Nitric Oxide, Blood Flow, Leg Ulcer, Sickle Cell Anemia, Thalassemia, Hemolytic Blood DisorderMar 2011Apr 2013
Study of Panobinostat (LBH589) in Patients With Sickle Cell DiseaseAbdullah Kutlarsickle cell anemia, sickle cell thalassemia, HDAC inhibitor, hemoglobin F, sickle beta thalassemiaNov 2010Dec 2014
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia MajorUniversity of WashingtonBeta Thalassemia Major, Hematopoietic Stem Cell Mobilization, Gene Transfer TechniquesOct 2010Dec 2012
A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)Thomas Jefferson UniversitySickle Cell Anemia, Sickle Cell Disease, Hemoglobin SS Disease, Hemoglobin S beta-0 Thalassemia, Inflammation, Quality of Life, Sickle Thalassemia, C-Reative Protein, Hemolytic Anemia, Hemostasis, Biomarkers, Coagulation, Omega-3 Fatty Acids, Eicosapentaenoic Acid, Docosahexaenoic Acid, Fish Oils, Drug: Placebo, Drug: LOVAZAOct 2010Mar 2012
B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic IndividualsAghia Sophia Children's Hospital of AthensOct 2010Feb 2012
Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationEmory Universitybone marrow transplant, sickle cell disease, thalassemia, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency, Schwachman-Diamond syndrome, Diamond-Blackfan anemia, Fanconi anemia, dyskeratosis-congenita, Chediak-Higashi syndrome, severe aplastic anemiaSep 2010Sep 2015
GDF 15 in Sickle Cell Disease and Hereditary SpherocytosisWolfson Medical CenterSep 2010Sep 2011
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron OverloadShire Development LLCBeta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelationAug 2010Jan 2013
Genes Influencing Iron Overload StateSt. Jude Children's Research HospitalIron overload, Blood TransfusionAug 2010Jul 2016
Safety and Efficacy of Deferasirox in β-thalassaemia Patients With Severe Cardiac Iron OverloadNovartis PharmaceuticalsSevere cardiac iron overload, deferasirox, β-thalassaemia, cardiac dysfunctionJul 2010Dec 2014
Sickle Cell Trait and the Risk of Venous ThromboembolismAlbert Einstein College of Medicine of Yeshiva Universitysickle cell trait, thrombosis, D-DimerJul 2010Dec 2012
Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell DiseaseUniversity of Miamisickle cell disease, propanolol, red blood cell adhesionJun 2010Dec 2016
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemiaEmmaus Medical, Inc.Sickle Cell Anemia, Sickle Cell Pain Crises, Painful Crises, Sickle Cell Pain, Vaso-occlusive CrisesMay 2010Feb 2013
Perceptions of Thalassemia Major in Singapore: An Exploratory Study of StigmaNational Human Genome Research Institute (NHGRI)Thalassemia Major, Stigma, Singapore, QualitativeApr 2010
Beta-thalassemia and MicroparticlesAssistance Publique Hopitaux De MarseilleTM, TI, MPMar 2010Mar 2013
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study (THALASSA)Novartis PharmaceuticalsThalassemia,, thalassemia intermedia,, alpha-thalassemia,, beta-thalassemia,, deferasirox,, iron overload,, non-transfusion dependentMar 2010Jun 2012
Multi-Center Study of Iron Overload: Survey Study (MCSIO)Children's Hospital & Research Center OaklandMar 2010Oct 2013
Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron OverloadHormozgan University of Medical SciencesThalassemia, Iron overload, transfusion, osveral, desferalFeb 2010May 2011
Reduced Intensity Transplant Conditioning Regimen for Severe ThalassemiaWashington University School of MedicineThalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablativeJan 2010Sep 2011
This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.Novartis PharmaceuticalsIron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron OverloadDec 2009Sep 2011
Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major PatientsTehran University of Medical SciencesThalassemia, BMT, HSCTNov 2009Dec 2011
Multi-Center Study of Iron Overload: Pilot StudyChildren's Hospital & Research Center OaklandNov 2009Sep 2012
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron OverloadFerroKin BioSciences, Inc.Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelationNov 2009Dec 2010
Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern IsraelHaEmek Medical Center, IsraelOct 2009Dec 2010
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-ThalassemiaNational Heart, Lung, and Blood Institute (NHLBI)Peripheral Blood Stem Cell Transplantation, Anemia, Sickle Cell, Graft-Versus-Host Disease, Sirolimus (Rapamune(Registered Trademark)), Alemtuzumab (Campath(Registered Trademark)), Thalassemia, Sickle Cell DiseaseSep 2009Aug 2015
Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood TransfusionsNational Heart, Lung, and Blood Institute (NHLBI)Transfusion-Dependant Thalassemia, Pain, Mild PainJul 2009Oct 2010
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological DisordersSingapore General HospitalChronic cytopenic haematological diseases, Chinese herbal concoction, Supportive managementJul 2009Aug 2011
Treosulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersFred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumNonmalignant diseases, nonmalignant inherited disorders, primary immunodeficiency diseases, primary immune deficiency disorders, Chronic granulomatous disease, IPEX syndrome, hemophagocytic lymphohistiocytosis, Wiskott Aldrich Syndrome, bone marrow failure syndromes, Shwachman Diamond Syndrome, Dyskeratosis Congenita, Diamond Blackfan Anemia, inborn errors of metabolism, metabolic diseases, hemoglobinopathies, sickle cell disease, thalassemia, reduced intensity transplantation, hematopoietic cell transplantation, bone marrow transplantation, umbilical cord blood transplantationJul 2009Jul 2017
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell DiseaseUniversity of PennsylvaniaJul 2009Aug 2012
Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E PatientsMahidol UniversityThalassemia, Oxidative stress, antioxidant cocktailJun 2009Jun 2011
Blood Transfusions in Thalassemia Patients, Complications and Adverse EffectsHaEmek Medical Center, IsraelBlood transfusions, Adverse events, Hepatitis C, HIVMay 2009Nov 2010
Phase 1/2 Study of HQK-1001 in Patients With Beta ThalassemiaHemaQuest Pharmaceuticals Inc.Mar 2009May 2010
Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary HypertensionNational Heart, Lung, and Blood Institute (NHLBI)Pulmonary HypertensionMar 2009Nov 2010
Therapeutic Effects of Silymarin in Patients With B-thalassemia MajorIsfahan University of Medical Sciencesiron overload condition, regular desferrioxamine administration, receiving continuous blood transfusionsMar 2009Dec 2009
Assessment of Pain in People With ThalassemiaNational Heart, Lung, and Blood Institute (NHLBI)Transfusion-Dependent Thalassemia, Non-Transfusion-Dependent ThalassemiaMar 2009Dec 2010
Glutamine Therapy for Hemolysis-Associated Pulmonary HypertensionChildren's Hospital & Research Center OaklandPulmonary Hypertension, Sickle Cell Disease, ThalassemiaMar 2009Mar 2013
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCsStanford UniversityMar 2009Aug 2013
The Spleen in Sickle Cell Anemia and Sickle Cell ThalassemiaHaEmek Medical Center, IsraelSplenectomy, Thrombocytosis, Infections, Spleen, Sickle Cell ThalassemiaFeb 2009Aug 2010
Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 MsecNovartis PharmaceuticalsThalassemia, cardiac MRI T2*Feb 2009May 2011
Iron Overload and Growth Velocity in Thalassemia and Sickle Cell AnemiaHaEmek Medical Center, IsraelThalassemia Major, Thalassemia Intermedia, Sickle Cell Anemia, Sickle Cell Thalassemia, Iron Overload, Growth VelocityJan 2009Jul 2010
Interferon and Ribavirin Treatment in Patients With HemoglobinopathiesAzienda Ospedaliera V. Cervelloantiviral treatment in hemoglobinopathies, management of chronic viral hepatitis C infection, antiviral treatmentJan 2009Dec 2013
European Sickle Cell Disease Cohort - HydroxyureaADDMEDICA SASAJan 2009Dec 2018
Fresh Versus Old Red Blood Cells for TransfusionColumbia Universityred blood cells, transfusion, storage, sickle cell disease, β-thalassemiaDec 2008Mar 2011
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced IntensityNorth Shore Long Island Jewish Health SystemReduced Intensity Conditioning RegimenDec 2008Jan 2017
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron OverloadNovartis PharmaceuticalsThalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependentNov 2008Jun 2011
Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia MajorUniversity of Campinas, Brazilthalassemia major, calcium channel blockade, magnetic resonance imagingSep 2008Jul 2011
Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell ThalassemiaHaEmek Medical Center, IsraelSickle Cell Anemia, Sickle Cell Thalassemia, Iron overload, Non transferrin binding iron, HepcidinSep 2008Dec 2010
Evaluation of Non-invasive Endothelial Function in Children Sickle by Vascular UltrasoundUniversity Hospital, Toulouseendothelial function, flow-mediated dilation, vascular elastic propertiesSep 2008Sep 2016
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsChildren's Hospital Los Angelesunrelated, BMT, HSCT, bone marrow transplantation, sickle cell disease, thalassemia, CGD, HLH, Blackfan-Diamond anemia, Hurler, leukodystrophy, LAD I, Genetic diseases, Red blood cell defects, Sickle cell disease, Thalassemia, Leukocyte defects and immune deficiencies, Hereditary Lymphohistiocytosis, chronic granulomatous disease, Wiskott-Aldrich syndrome, Chediak Higashi syndrome, CD40 ligand deficiency, Hyper IgM syndrome, leucocytes adhesion defect type 1, Osteopetrosis, congenital neutropenia, X-linked lymphoproliferative disease, Platelets defects, Congenital amegakaryocytic thrombocytopenia, Metabolic and storage disorders, Hurler disease, leukodystrophies, Niemann-Pick disease, Fucosidosis, Stem cell defects, reticular agenesisAug 2008Aug 2012
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant DisordersDuke UniversityImmunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, SCIDS, Wiskott Aldrich, FEL, HLH, IPEX, LAD, Sickle Cell, Thalassemia, Omenn's Syndrome, Hurler's Syndrome, MLD, ALD, Sanfilippo, Krabbe, Hunter's syndrome, TaySachs, Diamond Blackfan Anemia, transplant, MPS, GaucherAug 2008Dec 2013
Iron Balance Study of Deferasirox, Deferoxamine and the Combination of BothWeill Medical College of Cornell UniversityIron chelation, Iron balance, Secondary iron overload, deferoxamine, deferasiroxAug 2008Dec 2009
Oral Nifedipine to Treat Iron OverloadNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Nifedipine, Thalassemia, Hemochromatosis, Iron Overload, Iron ExcretionJun 2008Sep 2010
Sickle Cell Anemia Screening and Prevention in Northern IsraelHaEmek Medical Center, IsraelThalassemia, Sickle cell anemia, Carrier detection, Prenatal Diagnosis, Genetic counselingMay 2008
Clinical Diagnosis of Teenagers Admitted to Pediatric DepartmentsHaEmek Medical Center, IsraelMay 2008
Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With FeverHaEmek Medical Center, IsraelFever, Blood culturesMay 2008Nov 2008
Diabetic Retinopathy and Sickle TraitMedical University of South CarolinaDiabetic Retinopathy, Sickle Cell Trait, Vision Loss, Eye diseaseMay 2008Jul 2010
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia MajorMemorial Sloan-Kettering Cancer CenterGCSFApr 2008Feb 2011
Pilot Study for Patients With Poor Response to DeferasiroxChildren's Hospital BostonThalassemia, Iron, ChelationMar 2008Nov 2008
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia IntermediaNational Heart, Lung, and Blood Institute (NHLBI)Thalassemia IntermediaJan 2008Sep 2010
MRI Evaluation of Iron Overload in the Heart, Liver and Pancreas in Patients Receiving Multiple Blood Transfusions.Sheba Medical Centeriron overload, Thalassemia, Anemia, Sickle Cell, Magnetic Resonance ImagingJan 2008May 2010
Far Infrared Radiation for Sickle Cell Pain ManagementGAAD Medical Research Institute Inc.HEMOGLOBIN SC DISEASE, HEMOGLOBIN C DISEASE, SICKLE CELL TRAIT, THALASSEMIAJan 2008Jan 2009
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron OverloadNovartis PharmaceuticalsMyelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasiroxDec 2007Jul 2011
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron OverloadNovartis Pharmaceuticalsiron overload, cardiac iron, haemosiderosis, myocardial T2*, left ventricular ejection fraction, LVEF, cardiac dysfunction, thalassaemia, Diamond Blackfan anemia, DBA, sideroblastic anemia, myelodysplastic syndromes, MDS (low and INT-1 risk as per the IPSS for MDS), liver MRI, deferasirox, deferoxamine, ICL670, DFO, cardiovascular magnetic resonance imagingNov 2007Mar 2013
Blood Sampling for Research Related to Sickle Cell DiseaseNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Sickle Hemoglobin, Erythrocytes, Drug Screen, Sickle Cell Trait, Sickle Cell DiseaseOct 2007
Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell ThalassemiaHaEmek Medical Center, IsraelSickle Cell Anemia, Sickle Cell Thalassemia, Iron Overload, MRISep 2007Dec 2010
Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron OverloadNovartis Pharmaceuticalsβ-thalassemia, transfusional iron overload, renal function, renal biomarkers, deferasiroxSep 2007Sep 2012
Combo Chelation TrialChildren's Hospital & Research Center OaklandThalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overloadSep 2007Oct 2012
Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or ThalassemiaSt. Jude Children's Research HospitalJul 2007Jul 2016
Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia PatientsChina Medical University HospitalJul 2007Jul 2008
Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia IntermediaShiraz University of Medical SciencesHydroxyurea, magnesium chloride, L-carnitine, β-thalassemia intermediaJun 2007Dec 2007
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) StudyNew England Research InstitutesMay 2007Jun 2011
Endocrine and Nutritional Assessment in B Thalassemia MajorHaEmek Medical Center, IsraelThalassemia, Nutrition, Endocrine, BMIMay 2007Dec 2009
Pegasys® Plus Ribavirin in Thalassemic Patients With Hepatitis C Virus InfectionBaqiyatallah Medical Sciences UniversityHepatitis C, Thalassemia, Pegasys, RibavirinMay 2007Sep 2009
Long Term Follow up in Sickle Cell Patients Treated by HydroxyureaHaEmek Medical Center, IsraelSickle cell anemia, Sickle cell thalassemia, HydroxyureaMay 2007Dec 2008
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant DisordersCelgene CorporationMyelodysplastic Syndrome (MDS), Acute myelogenous Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Sickle Cell Disease (SCD), Beta Thalassemia, Inborn Errors of Metabolism, Severe Combined Immunodeficiency Disease (SCID)May 2007Dec 2013
Sickle Cell Anemia in an Arab Bedouin Village in the Northern IsraelHaEmek Medical Center, IsraelSickle cell disease, Sickle cell thalassemia, Population screeningMay 2007Dec 2008
Detection of β Thalassemia Carriers by Red Cell Parameters Obtained From the H2 Automatic CounterHaEmek Medical Center, IsraelThalassemia, Iron Deficiency, Carrier screeningMar 2007Jun 2007
T2* in Transfusion Dependant Anemia, MI, LVF, Normal PatientsImperial College LondonJan 2007Aug 2007
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell DiseaseSt. Jude Children's Research HospitalSickle Cell Disease, Vaso-occlusive Event, Painful Crises, Acute Chest SyndromeJan 2007Aug 2009
Autologous Bone Marrow Stem Cell Transplantation for Hip Osteonecrosis in Sickle Cell DiseaseFederal University of BahiaCell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cellAug 2006Aug 2015
Autologous Bone Marrow Stem Cells for Chronic Leg Ulcer Treatment in Sickle Cell DiseaseFederal University of BahiaCell Therapy, Sickle cell disease, Autologous implantation, Mesenchymal stromal cell, Edothelial Progenitor cellAug 2006Aug 2016
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia MajorUniversity of WashingtonBeta Thalassemia Major, Hematopoietic Stem Cell Mobilization, Gene Transfer TechniquesJul 2006Aug 2010
Cardiopulmonary Function Assessment and NO-Based Therapies for Patients With Hemolysis-Associated Pulmonary HypertensionNational Heart, Lung, and Blood Institute (NHLBI)Nitric Oxide, PDE Inhibitors, Pulmonary Hypertension, Sickle Cell, Thalassemia, Sickle Cell AnemiaJul 2006Mar 2010
Efficacy and Safety of Deferasirox in Patients With Transfusion-Dependent Iron OverloadNovartis PharmaceuticalsThalassemia, Myelodysplastic Syndrome, MDS, Sickle cell, Diamond-Blackfan anemia, Transfusion, Anemia, Fanconi, Chelation, DeferasiroxJul 2006May 2009
The Change of Coagulation Markers in Children With β-Thalassemia Disease After Stem Cell TransplantationMahidol UniversityCoagulation markers, thalassemia disease post SCTJun 2006Dec 2009
Zinc & Bone Health in Thalassemia: The Think Zinc StudyChildren's Hospital & Research Center Oaklandzinc, thalassemia, bone mineral densityApr 2006Feb 2011
Compassionate Use of Deferiprone for Patients With Thalassemia and Iron-Induced Heart DiseaseChildren's Hospital of PhiladelphiaIron overload, Thalassemia, Iron induced heart disease, deferoxamine (Desferal), DeferiproneMar 2006
Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGRObstetrix Medical GroupNIH, Severe Symmetrical IUGR, Tandem Mass Spectrometry, Nonimmune Hydrops, Severe Symmetrical Intrauterine Growth RestrictionMar 2006Aug 2009
Cardiac T2* in Beta-thalassemia Patients on Deferasirox TreatmentNovartisIron Chelation, Deferasirox, Chelator, Desferal, beta-thalassemia, Iron overloadFeb 2006Nov 2009
Invasive Infections in Children With HemoglobinopathiesHaEmek Medical Center, IsraelThalassemia, Sickle cell anemia, Splenectomy, Infections, Streptococcal PneumoniaJan 2006Jun 2006
Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria ResearchNational Institute of Allergy and Infectious Diseases (NIAID)Sickle, Hemoglobinopathy, Thalassemia, G6PD, Plasmodium, Erythrocyte Variant, Hemoglobin Variant, Healthy Volunteer, HVJan 2006Jul 2010
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron OverloadNovartis PharmaceuticalsDeferasirox, Congenital Anemias, Anemias, Red Blood Cell Disorders, Chronic Iron Overload, Transfusional Iron Overload, Iron Chelators, Oral Iron Chelators, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis, ICL670AOct 2005Oct 2008
Examination of Protective Factors Against Severe MalariaNational Institute of Allergy and Infectious Diseases (NIAID)Sickle Trait, Fetal Hemoglobin, Hemoglobin C, Beta-Thalassemia, Artesunate-AmodiaquineAug 2005Apr 2009
Factors Promoting Increased Rate and Success of Pregnancy in the Thalassemia Population in TorontoUniversity Health Network, TorontoJul 2005
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOPBaylor College of MedicineSickle Cell Disease, SCD, Hemoglobin SS, Hemoglobin SC, Hemoglobin Sb0/+, HLA genotype, Severe anemia, Transfusion therapyJul 2005Jul 2015
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)Novartis PharmaceuticalsTransfusional hemosiderosis, Beta-thalassemia major, Deferasirox, iron overload, rare anemia, iron overload due to transfusionJun 2005May 2008
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac DiseasesNational Heart, Lung, and Blood Institute (NHLBI)Jun 2005Apr 2009
Heart Disease in Sickle Cell AnemiaNational Institutes of Health Clinical Center (CC)Cardiac Arrhythmias, Diastolic Dysfunction, Pulmonary Hypertension, Sickle Cell Anemia, Sudden Death, ThalassemiaJun 2005Oct 2007
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron OverloadNovartis PharmaceuticalsThalassemia, Myelodysplastic Syndrome, MDS, Sickle cell, Diamond-Blackfan anemia, Transfusion, Anemia, Fanconi, Chelation, DeferasiroxApr 2005Jun 2010
Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent PatientsLipomedDeferiprone, L1, Desferrioxamine, Hemochromatosis, Iron overload, ThalassemiaMar 2005May 2011
Aspirin Prophylaxis in Sickle Cell DiseaseUniversity of Rochestersickle cell disease, hemoglobin SS disease, hemoglobin S Beta-0 Thalassemia, silent infarction in sickle cell disease, overt stroke in sickle cell disease, aspirin, transcranial Doppler ultrasound, neurocognitive testingMar 2005Nov 2009
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or ThalassemiaNational Institute of Allergy and Infectious Diseases (NIAID)Jan 2005Dec 2005
An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron OverloadNovartis Pharmaceuticalsβ-thalassemia, iron overload, deferasiroxOct 2004Apr 2008
Genetic Factors Affecting the Severity of Beta ThalassemiaHadassah Medical OrganizationJul 2004Apr 2012
A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional HemosiderosisNovartisThalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiproneMay 2004Nov 2006
Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia MajorRoyal Brompton & Harefield NHS Foundation TrustRandomized Controlled Trial, Deferiprone, Deferoxamine, Iron chelation, Beta Thalassemia MajorMay 2004Jun 2005
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia MajorDana-Farber Cancer Institutechimerism, allogenic hematopoietic transplantation, assayApr 2004Oct 2005
Genetics of Alpha Thalassemia in Israeli Ethnic GroupsHadassah Medical OrganizationApr 2004Apr 2012
Natural History of Sickle Cell Disease and Other Hemolytic DisordersNational Heart, Lung, and Blood Institute (NHLBI)Acute Chest Syndrome, Hemoglobin, Nitric Oxide, Pulmonary Hypertension, Pain Crisis, Treatment Options, Sickle Cell Disease, Sickle Cell Trait, Red Blood Cell Disorder, Thalassemia, Paroxysmal Nocturnal Hemoglobinuria, Hemolytic DisordersApr 2004
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 ThalassemiaEmmaus Medical, Inc.sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous), Sickle ß0-ThalassemiaMar 2004Jul 2008
Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal EventsNovartis Pharmaceuticalsthalassemia, zoledronic acid, skeletal eventsMar 2004Mar 2006
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With HemoglobinopathiesDana-Farber Cancer InstituteHemoglobinopathies, Sickle cell anemia, sickle cell-hemoglobin C disease, sickle cell-B-thalassemia, transfusion-dependant thalassemia, allogeneic transplant, nonmyeloablative transplant, Stem cell transfusion, graft vs. host diseaseMar 2004Jul 2009
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia PatientsNovartis Pharmaceuticalsβ-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosisMar 2004Oct 2008
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLOBaylor College of Medicinetransfusion-dependent, homozygous b0/+-thalassemia, severe variants of b0/+-thalassemia, chronic transfusion therapy, iron chelating agents, severe, transfusion-dependent homozygous b0/+-thalassemia or severe variants of b0/+-thalassemiaFeb 2004Sep 2015
Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TIEnte Ospedaliero Ospedali GallieraThalassemia, Neridronate, Osteopenia, OsteoporosisJan 2004May 2010
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-ThalassemiaGenzymeBeta ThalassemiaSep 2003
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.Novartis Pharmaceuticalsβ-thalassemia major, iron overload, deferasirox, pediatric rare anemiaSep 2003Feb 2008
A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot StudyNational Heart, Lung, and Blood Institute (NHLBI)Sickle Cell Disease, Sickle Cell, Memory, Learning, AchievementSep 2003Dec 2012
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe ToxicityNovartis PharmaceuticalsDeferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional hemosiderosis, Congenital aplastic anemia (Diamond Blackfan anemia), Red cell aplasia, Thalassemia, β thalassemiaJun 2003Dec 2007
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood TransfusionsNovartis PharmaceuticalsThalassemia, iron overload, deferoxamine, hemosiderosisMay 2003Nov 2004
Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study)National Heart, Lung, and Blood Institute (NHLBI)May 2003Aug 2006
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic AnemiasNovartis Pharmaceuticalsbeta-thalassemia, iron overload, deferoxamine, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, AplasticMay 2003Nov 2004
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital AnemiasNational Heart, Lung, and Blood Institute (NHLBI)Stem Cell Transplant, Low Dose Radiation, Alemtuzumab (Campath), Sirolimus (Rapamune), Donor Apheresis, Graft-Versus-Host Disease, Graft-Versus-Marrow, Host-Donor Chimerism, Peripheral Blood Stem Cells, Low Dose Irradiation, Sickle Cell Anemia, SCA, Thalassemia, Diamond-Blackfan Anemia, DBAMay 2003Feb 2018
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension StudyNovartis Pharmaceuticalsiron overload, iron chelation therapy, B-thalassemiaFeb 2003Jan 2008
L-Glutamine Therapy for Sickle Cell AnemiaLos Angeles Biomedical Research Institutesickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous and heterozygous), Sickle ß0-ThalassemiaJan 2003Nov 2009
Efficacy Study in Removing Excess Iron From the HeartApoPharmaIron Overload, Thalassemia, Haemosiderosis, Cardiac, Deferiprone, ChelationDec 2002Oct 2004
A Pilot Study of HSCT for Patients With High-Risk Hemoglobinopathy Using a Nonmyeloablative Preparative RegimenUniversity of PittsburghSickle Cell Disease, Thalassemia, HemoglobinopathiesJun 2002Dec 2012
Stem Cell Transplant for HemoglobinopathyMasonic Cancer Center, University of Minnesotahigh risk hemoglobinopathy, stem cell transplant, donor lymphocyte infusion, transfusion dependent, stem cell donor, cord blood, marrow, transfusion dependent non-malignant hematologic disordersJun 2002Jun 2016
Study With Deferiprone and/or Desferrioxamine in Iron Overloaded PatientsLipomedDeferiprone, L1, Desferrioxamine, Hemochromatosis, Iron overload, ThalassemiaJan 2002
Stem Cell Transplant in Sickle Cell Disease and ThalassemiaColumbia Universitystem cell transplant, sickle cell disease, thalassemia, moderately ablative, cord blood transplant, matched family donorDec 2001Jan 2014
Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia MajorOspedale MicrocitemicoDec 2001Jun 2006
Oral Magnesium Pidolate, Hemoglobin SC Disease, MG PidolateBaylor College of MedicineHemoglobin SC disease, sickle cell-hemoglobinJan 2001May 2006
Phase II Study of Azacitidine and Phenylbutyrate in Patients With Thalassemia MajorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, thalassemia majorNov 2000
Efficacy Study of the Use of Sequential DFP-DFO Versus DFPAzienda Ospedaliera V. Cervellothalassemia major, chelation treatment, secondary hemochromatosisSep 2000Jan 2008
Arginine Therapy in Sickle Cell Disease-VOC Clinical TrialChildren's Hospital & Research Center Oaklandsickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxideSep 2000Jun 2007
Bone Marrow Transplant From Related Donor for Patients With High Risk HemoglobinopathiesBaylor College of MedicineAug 2000
Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk HemoglobinopathiesBaylor College of MedicineAug 2000
Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)National Heart, Lung, and Blood Institute (NHLBI)chelator, iron, transfusion, anemia, thalassemiaJul 2000Jul 2006
5-Azacytidine and Phenylbutyrate to Treat Severe ThalassemiaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Fetal Hemoglobin, 5-Azacytidine, Phenylbutyrate, Thalassemia, Hemoglobin, Anemia, Blood, Thalassemia Major, Beta ThalassemiaJun 2000Jun 2003
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic DisordersFairview University Medical Centerchronic congenital neutropenia, chronic neutropenia, congenital pure red cell aplasia, disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, neutropenia, pure red cell aplasia, rare disease, sickle cell anemia, thalassemia majorJun 2000
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling DisordersNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Fetal Hemoglobin, Hematopoietic Growth Factor, Peripheral Blood CD34 Cells, Vasoocclusive Crisis, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell DisorderMar 2000Oct 2000
Mobilization and Handling of Stem Cells for Transplant From Healthy Volunteers With Sickle Cell TraitNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Donor Apheresis, Sickle Cell Trait, Red Cell AlloimmunizationJan 2000Jul 2002
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Gamma Gene, Hemoglobin Switching, Erythropoiesis, HbE Hemoglobin Chain Synthesis Imbalance, Cooley's Anemia, Beta-Thalassemia IntermediaDec 1999Feb 2002
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia IntermediaBoston Universitygenetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, thalassemia intermediaMar 1999
Cord Blood Transplantation for Sickle Cell Anemia and ThalassemiaNational Heart, Lung, and Blood Institute (NHLBI)Jan 1999Aug 2006
Combination Iron Chelation TherapyNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)chelation therapy, combination chemotherapy, iron poisoning, thalassemia, chelating agent, deferoxamine, drug screening /evaluation, therapy evaluationDec 1998Nov 2002
Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patients With Thalassemia MajorNational Center for Research Resources (NCRR)disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, osteoporosis, rare disease, thalassemia majorJun 1998Aug 2000
Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell UlcersFDA Office of Orphan Products Developmentdermatologic disorders, genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, sickle cell anemia, skin ulcers, thalassemia majorSep 1997
Stem Cell Transplantation (SCT) for Genetic DiseasesNational Center for Research Resources (NCRR)Fanconi's anemia, amegakaryocytic thrombocytopenia, aplastic anemia, congenital pure red cell aplasia, genetic diseases and dysmorphic syndromes, hematologic disorders, inborn errors of metabolism, metachromatic leukodystrophy, pure red cell aplasia, rare disease, sphingolipidoses, thalassemia majorJan 1995
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of ErythropoiesisMemorial Sloan-Kettering Cancer CenterERYTHROPOIESIS, Genetic Disorders, Sickle Cell Anemia, Thalassemia, Diamond Blackfan AnemiaJan 1994Aug 2008
Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl HydrazoneNational Heart, Lung, and Blood Institute (NHLBI)Jun 1989
Deferoxamine for the Treatment of HemochromatosisNational Heart, Lung, and Blood Institute (NHLBI)Desferal, Thalassemia, Liver Iron Concentration, Endocrine Evaluation, Diabetes Mellitus, Cardiac Disease, Acquired AnemiaApr 1985
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional HemochromatosisNational Heart, Lung, and Blood Institute (NHLBI)Jan 1978
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood TransplantsJoanne KurtzbergHaploidentical Donor, T-cell depleted Stem Cells, Allogeneic Transplant, Umbilical Cord Blood Donor, High Risk Malignancies, Metabolic Disorders, Immune Deficiency, Acute Lympoblastic Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome, ALL, AML, MDS, CGD, SCID, Adrenoleukodystrophy, Metachromaticleukodystrophy, Krabbe, PMD, Hunter's, Hurler's, Severe Aplastic Anemia, Lymphoma, Sickle Cell Disease, Thalassemia
A Dose Escalation Study of Intravenous L-citrulline in Steady-state Sickle Cell DiseaseSuvankar Majumdar

Future events

  • Towards Transformative Therapies for Sickle Cell Disease
  • Ninth International Leaders in Biobanking Congress
  • 14th International Conference on Thalassaemia & Haemoglobinopathies & the 16th TIF International Conference for Patients & Parents
  • 5th Annual Advances in Prenatal Molecular Diagnostics
  • PGD and PGS: past, present and future. A celebration Campus event to mark the 20th anniversary of the PGD Consortium

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