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ClinicalTrials.gov public records Last synced May 21, 2026, 5:56 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 113 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Beta Thalassemia Clear all
Recruiting No phase listed Observational
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 May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 5:56 PM EDT
Active, not recruiting Phase 3 Interventional
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 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Thalassemia Major
Interventions
DST-0509, Jadenu, Exjade
Drug
Lead sponsor
DisperSol Technologies, LLC
Industry
Eligibility
8 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced May 21, 2026, 5:56 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions
HU-Go app
Other
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 25 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced May 21, 2026, 5:56 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2043
U.S. locations
6
States / cities
Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2025 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
FBS0701
Drug
Lead sponsor
FerroKin BioSciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2012 · Synced May 21, 2026, 5:56 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Hemoglobinopathies, Sickle Cell Disease, Thalassemia
Interventions
Busulfex, Fludarabine, Alemtuzumab, Stem Cell Transfusion
Drug · Procedure
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
6
States / cities
Atlanta, Georgia • Shreveport, Louisiana • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2013 · Synced May 21, 2026, 5:56 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia Major
Interventions
CliniMACS
Device
Lead sponsor
Timothy Olson
Other
Eligibility
2 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced May 21, 2026, 5:56 PM EDT
Active, not recruiting Not applicable Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions
HU-Go app
Other
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 23, 2024 · Synced May 21, 2026, 5:56 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers
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 May 21, 2026, 5:56 PM EDT
Withdrawn Phase 1Phase 2 Interventional
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 May 21, 2026, 5:56 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
Interventions
Broccosprouts® (Brassica Protection Products LLC) homogenate
Dietary Supplement
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 27, 2015 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional
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 20, 2018 · Synced May 21, 2026, 5:56 PM EDT
Enrolling by invitation Phase 2 Interventional
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 May 21, 2026, 5:56 PM EDT
Enrolling by invitation No phase listed Observational
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 May 21, 2026, 5:56 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional
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 May 21, 2026, 5:56 PM EDT
Recruiting Phase 1Phase 2 Interventional
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 May 19, 2026 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 3 Interventional
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 May 21, 2026, 5:56 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
Interventions
IMR-687, Placebo Oral Capsule
Drug
Lead sponsor
Cardurion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated May 14, 2025 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Skin Ulcers, Sickle Cell Anemia
Interventions
Arginine Butyrate, Standard local care dressing
Drug · Other
Lead sponsor
Susan P. Perrine
Other
Eligibility
16 Years to 60 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2005
U.S. locations
4
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced May 21, 2026, 5:56 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Ambrisentan, Placebo
Drug
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 1, 2020 · Synced May 21, 2026, 5:56 PM EDT