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ClinicalTrials.gov public records Last synced Nov 2, 2026, 4:38 PM EST

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Showing 1–24 of 115 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Beta-Thalassemia Clear all
Recruiting Phase 2 Interventional

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

NCT04143724
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ACE-536
Drug
Lead sponsor
Celgene
Industry
Eligibility
6 Years to 17 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2035
U.S. locations
2
States / cities
Los Angeles, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Recruiting Phase 1Phase 2 Interventional

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338
View directory record Official record
Conditions
Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions
CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
Biological · Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 4 Interventional Results available

Pilot Study for Patients With Poor Response to Deferasirox

NCT00749515
View directory record Official record
Conditions
Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
Interventions
Deferoxamine, Deferasirox, HIDA
Drug · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 12, 2024 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

NCT01049854
View directory record Official record
Conditions
Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
Interventions
Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi)
Drug
Lead sponsor
New York Medical College
Other
Eligibility
Up to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 21, 2018 · Synced Nov 2, 2026, 4:38 PM EST
Terminated Phase 2 Interventional

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

NCT00034528
View directory record Official record
Conditions
Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
Interventions
Busulfan, Fludarabine, FK506, Prednisone
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 1, 2013 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional Results available

Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

NCT00661726
View directory record Official record
Conditions
Thalassemia
Interventions
Decitabine (USAN, INN)
Drug
Lead sponsor
Carelon Research
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2014 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional Results available

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

NCT00873041
View directory record Official record
Conditions
Non-transfusion Dependent Thalassemia
Interventions
deferasirox, placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 9, 2013 · Synced Nov 2, 2026, 4:38 PM EST
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Nov 2, 2026, 4:38 PM EST
Not listed Phase 4 Interventional

The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study

NCT01323608
View directory record Official record
Conditions
Thalassemia Major
Interventions
Vitamin D3, Placebo
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
6 Years to 60 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 19, 2011 · Synced Nov 2, 2026, 4:38 PM EST
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

NCT00001958
View directory record Official record
Conditions
Beta Thalassemia, Hemoglobinopathy
Interventions
Hydroxyurea
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Nov 2, 2026, 4:38 PM EST
Completed Not applicable Interventional Results available

Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

NCT00658385
View directory record Official record
Conditions
Beta Thalassemia Major, Congenital Anemias
Interventions
GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
Genetic
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 25, 2016 · Synced Nov 2, 2026, 4:38 PM EST
Terminated Phase 2 Interventional Results available

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

NCT01363908
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-Thalassemia
Interventions
SPD602
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Nov 2, 2026, 4:38 PM EST
Terminated Phase 1 Interventional

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

NCT07431398
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pociredir
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Miami, Florida • Orlando, Florida • Riverdale, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Nov 2, 2026, 4:38 PM EST
Recruiting No phase listed Observational Accepts healthy volunteers

Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

NCT04416178
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
13 Years and older
Enrollment
352 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Not listed Phase 2 Interventional

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

NCT00408447
View directory record Official record
Conditions
Sickle Cell Disease, Beta Thalassemia
Interventions
Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant
Drug · Procedure
Lead sponsor
Columbia University
Other
Eligibility
1 Month to 30 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Nov 2, 2026, 4:38 PM EST
Enrolling by invitation No phase listed Observational

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

NCT06664541
View directory record Official record
Conditions
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 29, 2024 · Synced Nov 2, 2026, 4:38 PM EST
Temporarily Not Available No phase listed Expanded access

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT07640815
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 55 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional Results available

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

NCT00447694
View directory record Official record
Conditions
Beta-thalassemia, Iron Overload
Interventions
Deferasirox
Drug
Lead sponsor
Novartis
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
3
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Nov 2, 2026, 4:38 PM EST
Completed Not applicable Interventional

Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

NCT04678037
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
Interventions
PROs assessment using sickle cell disease mobile app (SCD-app)
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 25 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Nov 2, 2026, 4:38 PM EST
Active, not recruiting Phase 3 Interventional

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05356195
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 11 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Nov 2, 2026, 4:38 PM EST
Completed Phase 2 Interventional

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
arginine butyrate, epoetin alfa
Drug
Lead sponsor
Boston University
Other
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
6
States / cities
Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2005 · Synced Nov 2, 2026, 4:38 PM EST