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ClinicalTrials.gov public records Last synced Oct 9, 2026, 10:49 AM EDT

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Showing 97–115 of 115 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Thalassemia, Beta Clear all
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 20, 2024 · Synced Oct 9, 2026, 10:49 AM 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 9, 2026, 10:49 AM 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 27, 2026 · Synced Oct 9, 2026, 10:49 AM EDT
Completed Phase 2 Interventional Results available

Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

NCT00004412
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 31, 2015 · Synced Oct 9, 2026, 10:49 AM EDT
Recruiting Phase 2 Interventional

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

NCT05735717
View directory record Official record
Conditions
Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, AML, TP53, Intrachromosomal Amplification of Chromosome 21, Cytogenetic Abnormality, CNS Leukemia, Minimal Residual Disease, Myelodysplasia, Juvenile Myelomonocytic Leukemia, Somatic Mutation, PTPN11 Gene Mutation, N-RAS Gene Amplification, Neurofibromatosis 1, NF1 Mutation, CBL Gene Mutation, Monosomy 7, Chromosome Abnormality, Fetal Hemoglobin, Lymphoblastic Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult
Interventions
Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam, Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells, Thymoglobulin, Cyclophosphamide
Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 60 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 9, 2026, 10:49 AM 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 23, 2026 · Synced Oct 9, 2026, 10:49 AM EDT
Completed Phase 2 Interventional

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

NCT03802201
View directory record Official record
Conditions
β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 16, 2021 · Synced Oct 9, 2026, 10:49 AM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 9, 2026, 10:49 AM EDT
Active, not recruiting Phase 2 Interventional

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

NCT06421636
View directory record Official record
Conditions
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions
REGN7999, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 9, 2026, 10:49 AM 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 9, 2026, 10:49 AM EDT
Completed Phase 1 Interventional

The Role of Endothelin-1 in Sickle Cell Disease

NCT02712346
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 2, 2020 · Synced Oct 9, 2026, 10:49 AM 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 8, 2019 · Synced Oct 9, 2026, 10:49 AM 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 3, 2025 · Synced Oct 9, 2026, 10:49 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

NCT00005893
View directory record Official record
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 9, 2026, 10:49 AM 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 7, 2024 · Synced Oct 9, 2026, 10:49 AM EDT
Completed Not applicable Interventional Results available

Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

NCT00658385
View directory record Official record
Conditions
Beta Thalassemia Major, Congenital Anemias
Interventions
GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
Genetic
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 25, 2016 · Synced Oct 9, 2026, 10:49 AM 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 4, 2014 · Synced Oct 9, 2026, 10:49 AM 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 16, 2025 · Synced Oct 9, 2026, 10:49 AM 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 22, 2017 · Synced Oct 9, 2026, 10:49 AM EDT