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ClinicalTrials.gov public records Last synced Oct 8, 2026, 10:28 AM EDT

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

Showing 97–111 of 111 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Beta Thalassemia-Major Clear all

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

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: 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

Conditions: Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions: Stem Cell Transplantation; Procedure
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 0 Years to 17 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1995
U.S. locations: 1
States / cities: Los Angeles, California

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

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

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: 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: 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

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

Conditions: Sickle Cell Disease
Interventions: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a; Biological
Lead sponsor: David Williams; Other
Eligibility: 3 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 2
States / cities: Los Angeles, California • Boston, Massachusetts

Conditions: Beta-thalassemia, Iron Overload
Interventions: Deferasirox; Drug
Lead sponsor: Novartis; Industry
Eligibility: 10 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 3
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois

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

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: Thalassemia
Interventions: Decitabine (USAN, INN); Drug
Lead sponsor: Carelon Research; Other
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

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

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

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

Stem Cell Transplantation (SCT) for Genetic Diseases

Effect of Broccoli Sprouts Homogenate on SS RBC

Safety Study for Beta Thalassemia Subjects on PTG-300

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

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 of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

Open-Label Extension Study to Pioneer Study 6058-SCD-101

Gene Transfer for Sickle Cell Disease

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

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

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

Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia