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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Beta Thalassemia Intermedia Clear all
Not listed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

NCT00061568
View directory record Official record
Conditions
Congenital Hemolytic Anemia, Sickle Cell Disease
Interventions
Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 28, 2024 · Synced Jul 23, 2026, 10:48 PM EDT
Recruiting Phase 2 Interventional

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

NCT05736419
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions
Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
2 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
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 13, 2021 · Synced Jul 23, 2026, 10:48 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 Jul 23, 2026, 10:48 PM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

NCT04008368
View directory record Official record
Conditions
Myeloid Chimerism
Interventions
CliniMACS CD34 Reagent
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

NCT04171635
View directory record Official record
Conditions
MRI Scans
Interventions
Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
Radiation
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
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 8, 2013 · Synced Jul 23, 2026, 10:48 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 23, 2026, 10:48 PM EDT
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 9, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
Completed Phase 1 Interventional

Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease

NCT01522547
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
pomalidomide
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2019 · Synced Jul 23, 2026, 10:48 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 Jul 23, 2026, 10:48 PM EDT
Terminated Phase 1 Interventional

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

NCT02986698
View directory record Official record
Conditions
Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia, Fetal Hydrops, Alpha; Thalassemia, Thalassemia Major, Thalassemia Alpha, A-Thalassemia
Interventions
in utero hematopoietic stem cell transplantation
Biological
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Weeks to 26 Weeks
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2025 · Synced Jul 23, 2026, 10:48 PM EDT
Completed Phase 1 Interventional

A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders

NCT00005783
View directory record Official record
Conditions
Hemoglobin SC Disease, Sickle Cell Anemia
Interventions
Recombinant-methionyl human stem cell factor
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 10:48 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 Jul 23, 2026, 10:48 PM EDT
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 25, 2019 · Synced Jul 23, 2026, 10:48 PM EDT
Recruiting Phase 3 Interventional

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Charlotte, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT06363760
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2040
U.S. locations
16
States / cities
Oakland, California • Aurora, Colorado • New Haven, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Jul 23, 2026, 10:48 PM EDT
Enrolling by invitation Phase 2 Interventional

Open-Label Extension Study to Pioneer Study 6058-SCD-101

NCT07401823
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pociredir
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
8
States / cities
Little Rock, Arkansas • Los Angeles, California • Baton Rouge, Louisiana + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
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 28, 2024 · Synced Jul 23, 2026, 10:48 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

NCT01966367
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 13, 2025 · Synced Jul 23, 2026, 10:48 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 23, 2026, 10:48 PM EDT
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 2, 2025 · Synced Jul 23, 2026, 10:48 PM EDT
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 17, 2026 · Synced Jul 23, 2026, 10:48 PM EDT
Recruiting No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Jul 23, 2026, 10:48 PM EDT