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Showing 25–33 of 9 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Transfusion Dependent Beta Thalassemia Clear all
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 Jun 22, 2026 · Synced Jul 24, 2026, 5:04 AM 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 Jul 24, 2026, 5:04 AM 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 Jul 24, 2026, 5:04 AM 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 24, 2026, 5:04 AM EDT
Completed Phase 2 Interventional

Safety Study for Beta Thalassemia Subjects on PTG-300

NCT04054921
View directory record Official record
Conditions
β-thalassemia, Ineffective Erythropoiesis
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 15, 2021 · Synced Jul 24, 2026, 5:04 AM 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 Jul 24, 2026, 5:04 AM 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 28, 2015 · Synced Jul 24, 2026, 5:04 AM 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 Jul 24, 2026, 5:04 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894
View directory record Official record
Conditions
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
Interventions
EDIT-301
Genetic
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
18 Years to 35 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
7
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Jul 24, 2026, 5:04 AM EDT