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Showing 1–24 of 130 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: THALASSEMIA MAJOR Clear all
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 10, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 2 Interventional

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

NCT00061763
View directory record Official record
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2017 · Synced Sep 6, 2026, 6:27 PM EDT
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 7, 2005 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 2 Interventional Results available

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

NCT01186419
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
SPD602 (FBS0701, SSP-004184)
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 60 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 3 Interventional

Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

NCT00061750
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ICL670, deferoxamine
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
595 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
7
States / cities
Los Angeles, California • Oakland, California • Stanford, California + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 18, 2012 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 1Phase 2 Interventional

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

NCT00069862
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
Deferitrin (GT56-252), desferoxamine (DFO)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 4, 2015 · Synced Sep 6, 2026, 6:27 PM EDT
Terminated Not applicable Interventional Results available

Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

NCT00578344
View directory record Official record
Conditions
Sickle Cell Disease, Hemoglobin SC
Interventions
Busulfan, Campath 1H, Cyclophosphamide and MESNA
Drug · Biological
Lead sponsor
Tami D. John
Other
Eligibility
Up to 40 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Sep 6, 2026, 6:27 PM EDT
Active, not recruiting Phase 2 Interventional

Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

NCT03924401
View directory record Official record
Conditions
Graft Versus Host Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 20 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Wilmington, Delaware • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Sep 6, 2026, 6:27 PM EDT
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 18, 2011 · Synced Sep 6, 2026, 6:27 PM EDT
Recruiting Phase 1Phase 2 Interventional

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

NCT07599176
View directory record Official record
Conditions
Sickle Cell Disease, Beta-thalassemia
Interventions
Total Body Irradiation, Alemtuzumab, Abatacept, Research blood draw
Radiation · Drug · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 65 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
Recruiting Phase 1 Interventional

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

NCT06647979
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
Interventions
autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
Biological · Device
Lead sponsor
Daniel Bauer
Other
Eligibility
13 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 2, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
Completed No phase listed Observational

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

NCT00000623
View directory record Official record
Conditions
Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia, Osteoporosis, Iron Overload, Hypertension, Pulmonary
Interventions
Deferoxamine, Deferiprone, Arginine, Sildenafil, Decitabine
Drug
Lead sponsor
Carelon Research
Other
Eligibility
1 Year to 75 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2014 · Synced Sep 6, 2026, 6:27 PM EDT
Withdrawn Phase 2 Interventional

Allograft for Sickle Cell Disease and Thalassemia

NCT02038478
View directory record Official record
Conditions
Sickle Cell Disease and Thalassemia
Interventions
Donor Stem Cell Transplantation
Biological
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Sep 6, 2026, 6:27 PM EDT
Active, not recruiting Phase 2 Interventional

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

NCT05353647
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 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
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 20, 2020 · Synced Sep 6, 2026, 6:27 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

NCT00040469
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
Campath -1H, Dilantin, Busulfan, Cyclophosphamide
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 64 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 17, 2020 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 6, 2026, 6:27 PM EDT
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 3, 2008 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 2 Interventional

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

NCT04987489
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat tablets
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
Cerritos, California • Los Angeles, California • Oakland, California + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Sep 6, 2026, 6:27 PM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

NCT02105766
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
Interventions
Alemtuzumab, Sirolimus, Cyclophosphamide, Pentostatin, Radiotherapy, Filgrastim
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Phase 2 Interventional

Safety Study for Beta Thalassemia Subjects on PTG-300

NCT04054921
View directory record Official record
Conditions
β-thalassemia, Ineffective Erythropoiesis
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 15, 2021 · Synced Sep 6, 2026, 6:27 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Reproductive Capacity and Iron Burden in Thalassemia

NCT02308904
View directory record Official record
Conditions
THALASSEMIA MAJOR
Interventions
Blood Draw/Semen Exam, Retrospective data/Chart Review/Relevant Clinical Results, Pituitary MRI
Other
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
12 Years and older
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced Sep 6, 2026, 6:27 PM EDT
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 12, 2024 · Synced Sep 6, 2026, 6:27 PM EDT