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Showing 1–24 of 207 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Thalassemia Clear all
Terminated Phase 2 Interventional

Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

NCT00040417
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
FLUDARABINE, CAMPATH-IH, Total Body Irradiation, FK506, G-SCF (Granulocyte-colony stimulating factor)
Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 65 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 Jul 20, 2026, 6:58 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Screening for Alpha Thalassemia in Healthy Volunteers

NCT02692872
View directory record Official record
Conditions
Alpha Thalassemia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 39 Years
Enrollment
367 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Jul 20, 2026, 6:58 PM EDT
Not listed Phase 2 Interventional

A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)

NCT01202812
View directory record Official record
Conditions
Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
Interventions
Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules
Dietary Supplement · Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
10 Years to 19 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 24, 2010 · Synced Jul 20, 2026, 6:58 PM EDT
Recruiting Not applicable Interventional

Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia

NCT07064941
View directory record Official record
Conditions
Thalassemia
Interventions
Exercise
Behavioral
Lead sponsor
University of California, San Francisco
Other
Eligibility
14 Years to 40 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
2
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jul 14, 2025 · Synced Jul 20, 2026, 6:58 PM EDT
Recruiting Phase 1Phase 2 Interventional

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

NCT06364774
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ALS20
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 40 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Jul 20, 2026, 6:58 PM EDT
Recruiting No phase listed Observational

Study of the Role of Genetic Modifiers in Hemoglobinopathies

NCT05799118
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
Interventions
GWAS
Genetic
Lead sponsor
Cyprus Institute of Neurology and Genetics
Other
Eligibility
2 Years and older
Enrollment
30,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 19, 2024 · Synced Jul 20, 2026, 6:58 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 20, 2026, 6:58 PM EDT
Completed Phase 1 Interventional

Haploidentical Hematopoietic Stem Cell Transplantation

NCT02165007
View directory record Official record
Conditions
Sickle Cell-thalassemia Disease, Thalassemia
Interventions
peripheral blood stem cell graft that are CD34+ selected
Drug
Lead sponsor
Catherine Bollard
Other
Eligibility
Up to 22 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Jul 20, 2026, 6:58 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 Jul 20, 2026, 6:58 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 20, 2026, 6:58 PM EDT
Completed Phase 1 Interventional

Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders

NCT01316796
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell Disease, Chronic Hemolytic Disorders
Interventions
Sodium Nitrite Cream
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 21, 2018 · Synced Jul 20, 2026, 6:58 PM EDT
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 Jun 30, 2026 · Synced Jul 20, 2026, 6:58 PM EDT
Completed Phase 2 Interventional

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

NCT00578435
View directory record Official record
Conditions
Genetic Disorders, Sickle Cell Anemia
Interventions
Busulfan, Cyclophosphamide, BMD
Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 11, 2008 · Synced Jul 20, 2026, 6:58 PM EDT
Recruiting Phase 2 Interventional

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

NCT02675959
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Defibrotide
Drug
Lead sponsor
New York Medical College
Other
Eligibility
6 Months to 34 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Valhalla, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 22, 2025 · Synced Jul 20, 2026, 6:58 PM EDT
Completed Phase 2 Interventional Results available

Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

NCT02125877
View directory record Official record
Conditions
Chronic Iron Overload Due to Transfusion-dependant Anemias
Interventions
Deferasirox dispersible tablet, Defearisox film-coated tablet
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
Orange, California • Chicago, Illinois • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2017 · Synced Jul 20, 2026, 6:58 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major

NCT00480506
View directory record Official record
Conditions
Thalassemia Major
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2005
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 22, 2012 · Synced Jul 20, 2026, 6:58 PM EDT
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 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 20, 2026, 6:58 PM EDT
Completed No phase listed Observational

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

NCT05255445
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Jul 20, 2026, 6:58 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 20, 2026, 6:58 PM EDT
Completed No phase listed Observational

Screening Patients With Sickle Cell Disease for Kidney Damage

NCT02239016
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
4
States / cities
Atlanta, Georgia • Louisville, Kentucky • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2021 · Synced Jul 20, 2026, 6:58 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 Jul 20, 2026, 6:58 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 20, 2026, 6:58 PM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced Jul 20, 2026, 6:58 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 20, 2026, 6:58 PM EDT