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Showing 1–24 of 35 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Transfusion-dependent Beta-Thalassemia Clear all
Recruiting Phase 3 Interventional

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

NCT07506863
View directory record Official record
Conditions
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Interventions
Mitapivat Matched Placebo, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
3
States / cities
New York, New York • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 25, 2026, 7:17 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 Sep 22, 2026 · Synced Sep 25, 2026, 7:17 PM EDT
Terminated Not applicable Interventional Results available

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

NCT00578292
View directory record Official record
Conditions
Thalassemia
Interventions
Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 64 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 30, 2020 · Synced Sep 25, 2026, 7:17 PM 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 Jun 30, 2026 · Synced Sep 25, 2026, 7:17 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 2, 2026 · Synced Sep 25, 2026, 7:17 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 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 25, 2026, 7:17 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

The BENeFiTS Trial in Beta Thalassemia Intermedia

NCT04432623
View directory record Official record
Conditions
Beta Thalassemia Intermedia, Sickle Cell Disease
Interventions
Benserazide Only Product
Drug
Lead sponsor
Phoenicia BioScience
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Sep 25, 2026, 7:17 PM EDT
Terminated Phase 2 Interventional

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

NCT03381833
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LJPC-401
Drug
Lead sponsor
La Jolla Pharmaceutical Company
Industry
Eligibility
14 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Sep 25, 2026, 7:17 PM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

NCT04770779
View directory record Official record
Conditions
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Interventions
Placebo Matching Mitapivat, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
258 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
10
States / cities
Phoenix, Arizona • La Jolla, California • Oakland, California + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 25, 2026, 7:17 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

NCT03432364
View directory record Official record
Conditions
Transfusion Dependent Beta-thalassemia
Interventions
ST-400 Investigational product
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Sep 25, 2026, 7:17 PM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 25, 2026, 7:17 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

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 6, 2024 · Synced Sep 25, 2026, 7:17 PM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

NCT04770753
View directory record Official record
Conditions
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions
Placebo Matching Mitapivat, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
6
States / cities
La Jolla, California • Palo Alto, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 PM 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 15, 2021 · Synced Sep 25, 2026, 7:17 PM EDT
Completed Phase 2 Interventional Results available

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

NCT03342404
View directory record Official record
Conditions
Thalassemia
Interventions
Luspatercept, Placebo, Best Supportive Care (BSC)
Drug · Other
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 19, 2023 · Synced Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 PM EDT
Active, not recruiting No phase listed Observational

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

NCT02633943
View directory record Official record
Conditions
Transfusion-dependent Beta-Thalassemia
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2035
U.S. locations
6
States / cities
Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 PM EDT
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 17, 2023 · Synced Sep 25, 2026, 7:17 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 Sep 25, 2026, 7:17 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
4
States / cities
Palo Alto, California • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 25, 2026, 7:17 PM EDT