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These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 5, 2026, 9:36 PM EDT

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

Showing 25–41 of 41 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Transfusion Dependent Thalassemia Clear all

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

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

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

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

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

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

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

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: 4
States / cities: Atlanta, Georgia • New York, New York • Philadelphia, Pennsylvania + 1 more

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

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

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

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

Conditions: Transfusion Dependent Thalassemia
Interventions: Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs); Device
Lead sponsor: Terumo BCTbio; Industry
Eligibility: 12 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • New York, New York + 1 more

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

Conditions: Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 59 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 6
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 2 more

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

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

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

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

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

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

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

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

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

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)

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

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

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

The BENeFiTS Trial in Beta Thalassemia Intermedia

Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)

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

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies