Beta-Thalassemia Major — Clinical Trials

MONDO:001648649 active·25 recruitingLast updated: Mar 22, 2026, 9:17 PM

Trial Landscape

Beta-Thalassemia Major has an active clinical trial landscape, driven by drug treatment and biological therapy.

Recruiting

Active Trials

By Approach

Drug Treatment20

Biological Therapy11

Gene Therapy3

Procedure3

Medical Device2

18 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Early Phase 1

Phase 1

Phase 2

Phase 3

Phase 4

25 trials are observational or have no phase designation.

Sponsor Types

Academic/Other29Industry19NIH2

Top Sponsors

Bioray Laboratories

Bristol-Myers Squibb

Celgene

CorrectSequence Therapeutics Co., Ltd

Editas Medicine, Inc.

Genetix Biotherapeutics Inc.

Trial Locations

United States19 trials

China14 trials

Italy9 trials

Germany5 trials

Egypt4 trials

Canada4 trials

United Kingdom3 trials

Turkey (Türkiye)3 trials

Spain2 trials

Pakistan2 trials

Trials often recruit across multiple countries, so totals exceed the trial count.

50 Trials

Phase 3Recruiting

NCT05477563

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Sponsor:Vertex Pharmaceuticals IncorporatedFDA Approved Drug

United StatesStarted: Aug 2022Est. completion: Jun 2027Updated: Nov 2025

Phase 2Recruiting

NCT05736419

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Sponsor:Memorial Sloan Kettering Cancer Center

United StatesStarted: Feb 2023Est. completion: Feb 2027Updated: Mar 2026

Phase 2Recruiting

NCT04143724

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Sponsor:Celgene

United StatesStarted: Nov 2019Est. completion: Jun 2035Updated: Jan 2026

Phase 2Recruiting

NCT05693909

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Sponsor:Pharmacosmos A/S

DenmarkStarted: Sep 2023Est. completion: May 2028Updated: Sep 2025

Phase 2Recruiting

NCT04099966

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Sponsor:Mitchell Cairo

United StatesStarted: Apr 2021Est. completion: Dec 2027Updated: Aug 2025

Phase 2Recruiting

NCT06490627

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

Sponsor:National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

PakistanStarted: Apr 2024Est. completion: Apr 2026Updated: May 2025

Phase 1RecruitingGene Therapy

NCT06364774

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Sponsor:Children's Hospital of Philadelphia

United StatesStarted: Apr 2025Est. completion: Dec 2027Updated: Feb 2026

Phase 1Recruiting

NCT06647979

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

Sponsor:Daniel Bauer

United StatesStarted: Dec 2025Est. completion: Dec 2030Updated: Feb 2026

Phase 1RecruitingGene Therapy

NCT06308159

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

Sponsor:Lantu Biopharma

ChinaStarted: May 2024Est. completion: Aug 2027Updated: Nov 2025

Phase 1Recruiting

NCT03653338

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Sponsor:Paul Szabolcs

United StatesStarted: Aug 2018Est. completion: Aug 2027Updated: Aug 2025

Phase 3Active, Not Recruiting

NCT05356195

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Sponsor:Vertex Pharmaceuticals IncorporatedFDA Approved Drug

United StatesStarted: May 2022Est. completion: May 2026Updated: Mar 2026

Phase 2Active, Not Recruiting

NCT05567458

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Sponsor:Bristol-Myers Squibb

ChinaStarted: Oct 2022Est. completion: Aug 2026Updated: Aug 2025

Phase 2Active, Not Recruiting

NCT06490601

Long Term Beta Thalassemia Treatment: Findings From The Extension Period

Sponsor:National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

PakistanStarted: Apr 2022Est. completion: Jul 2025Updated: May 2025

Phase 2Active, Not Recruiting

NCT02105766

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Sponsor:National Heart, Lung, and Blood Institute (NHLBI)

United StatesStarted: Apr 2014Est. completion: Dec 2027Updated: Mar 2025

Phase 2Active, Not Recruiting

NCT00408447

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Sponsor:Columbia University

United StatesStarted: Sep 2004Est. completion: Feb 2025Updated: Jun 2024

Last updated over 18 months ago

Phase 1Active, Not Recruiting

NCT05357482

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Sponsor:National Heart, Lung, and Blood Institute (NHLBI)

United StatesStarted: May 2022Est. completion: Jan 2027Updated: Mar 2026

Phase 1Active, Not Recruiting

NCT05444894

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

Sponsor:Editas Medicine, Inc.

United StatesStarted: Apr 2022Est. completion: Dec 2025Updated: Apr 2025

Early Phase 1Active, Not Recruiting

NCT06065189

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

Sponsor:Children's Hospital of Fudan University

ChinaStarted: Nov 2023Est. completion: Sep 2025Updated: Feb 2025

Phase 4Active, Not Recruiting

NCT05891249

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

Sponsor:Bristol-Myers Squibb

IndiaStarted: Jun 2023Est. completion: Dec 2028Updated: Oct 2025

Active, Not Recruiting

NCT06913634

Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Sponsor:Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

ItalyStarted: May 2023Est. completion: Jun 2026Updated: Apr 2025

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 22, 2026 at 9:17 PM

Beta-Thalassemia Major — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

50 Trials

Open Trials37

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

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

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Active Trials12

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Long Term Beta Thalassemia Treatment: Findings From The Extension Period

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

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

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Other1