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ClinicalTrials.gov public records Last synced Oct 20, 2026, 4:09 PM EDT

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Showing 1–24 of 208 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Thalassemia Clear all
Completed Phase 3 Interventional Results available

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

NCT02604433
View directory record Official record
Conditions
Erythrocyte Transfusion, Beta-Thalassemia
Interventions
Luspatercept, Placebo
Drug · Other
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
336 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 18, 2023 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032
View directory record Official record
Conditions
Thalassemia Major With Severe Transfusional Iron Overload
Interventions
Deferasirox and deferiprone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2019 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 2 Interventional

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

NCT00005934
View directory record Official record
Conditions
Beta Thalassemia
Interventions
5-Azacytidine
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 20, 2026, 4:09 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 4, 2008 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension

NCT01048905
View directory record Official record
Conditions
Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
Interventions
L-Glutamine
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
4 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 20, 2026, 4:09 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, Who do Not Have a β0/β0 Genotype

NCT02906202
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
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 3 Interventional Results available

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

NCT00235391
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2011 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Not applicable Interventional

Use of Mobile Technology for Intensive Training in Medication Management

NCT02133560
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Video recording, Medication Administration Log, Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
5 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 25, 2017 · Synced Oct 20, 2026, 4:09 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 5, 2015 · Synced Oct 20, 2026, 4:09 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 3, 2026 · Synced Oct 20, 2026, 4:09 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 24, 2005 · Synced Oct 20, 2026, 4:09 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 21 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 Jul 27, 2026 · Synced Oct 20, 2026, 4:09 PM EDT
Recruiting No phase listed Observational

International Registry of Patients With Alpha Thalassemia

NCT04872179
View directory record Official record
Conditions
Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 20, 2026, 4:09 PM EDT
Active, not recruiting Phase 1 Interventional

Gene Transfer for Sickle Cell Disease

NCT03282656
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
3 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 20, 2026, 4:09 PM EDT
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 Oct 20, 2026, 4:09 PM EDT
Completed No phase listed Observational

Genes Influencing Iron Overload State

NCT01158794
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Marrow Aplasia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Oct 20, 2026, 4:09 PM EDT
Terminated Phase 1 Interventional

Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

NCT02274233
View directory record Official record
Conditions
Iron Overload, Beta-Thalassemia
Interventions
SP-420
Drug
Lead sponsor
Sideris Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 29, 2015 · Synced Oct 20, 2026, 4:09 PM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

NCT07517133
View directory record Official record
Conditions
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions
Mitapivat, Placebo Matching Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
5
States / cities
Palo Alto, California • Atlanta, Georgia • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 20, 2026, 4:09 PM EDT
Completed Phase 4 Interventional

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

NCT03176849
View directory record Official record
Conditions
Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder, Pediatric Acute Myeloid Leukemia, Pediatric Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Sickle Cell Anemia in Children, Aplastic Anemia, Thalassemia in Children
Interventions
Vitamin D3, Standard Vitamin D3 Supplementation
Dietary Supplement
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
1 Year to 25 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 17, 2020 · Synced Oct 20, 2026, 4:09 PM EDT
Completed No phase listed Observational

Heart Disease in Sickle Cell Anemia

NCT00113152
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 60 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 4:09 PM EDT
Completed No phase listed Observational

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609840
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Thiotepa
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 20, 2026, 4:09 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Haploidentical PBMC Transplant for Severe Congenital Anemias

NCT00977691
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
PBSC Transplant, Alemtuzumab, Sirolimus, Cyclophosphamide, Low Dose Irradiation
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 20, 2026, 4:09 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 10, 2026 · Synced Oct 20, 2026, 4:09 PM EDT