Beta-Thalassemia Hbb/lcrb — Clinical Trials

MONDO:001351750 active·27 recruitingLast updated: Mar 19, 2026, 3:26 AM

Trial Landscape

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

Recruiting

Active Trials

By Approach

Drug Treatment17

Biological Therapy10

Gene Therapy7

Medical Device1

Procedure1

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/Other26Industry23NIH1

Top Sponsors

Agios Pharmaceuticals, Inc.

Bioray Laboratories

Bristol-Myers Squibb

Celgene

CorrectSequence Therapeutics Co., Ltd

Editas Medicine, Inc.

Trial Locations

United States22 trials

China20 trials

Italy11 trials

Greece8 trials

Germany7 trials

United Kingdom7 trials

Canada6 trials

Thailand6 trials

Turkey (Türkiye)6 trials

Malaysia5 trials

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

50 Trials

Phase 3Recruiting

NCT04064060

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Sponsor:Celgene

United StatesStarted: Aug 2019Est. completion: May 2028Updated: Nov 2025

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 IncorporatedOrphan Designation

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

Phase 2Recruiting

NCT06421636

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

Sponsor:Regeneron Pharmaceuticals

United StatesStarted: Sep 2024Est. completion: Jan 2028Updated: Mar 2026

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

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 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 IncorporatedOrphan Designation

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

Phase 3Active, Not Recruiting

NCT04770779

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

Sponsor:Agios Pharmaceuticals, Inc.

United StatesStarted: Nov 2021Est. completion: Jun 2029Updated: Mar 2026

Phase 3Active, Not Recruiting

NCT04770753

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

Sponsor:Agios Pharmaceuticals, Inc.

United StatesStarted: Dec 2021Est. completion: Dec 2028Updated: 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 1Active, Not Recruiting

NCT01966367

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sponsor:Diane George

United StatesStarted: Mar 2013Est. completion: Feb 2030Updated: Aug 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

Not ApplicableActive, Not Recruiting

NCT05860595

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

Sponsor:Institute of Hematology & Blood Diseases Hospital, China

ChinaStarted: May 2023Est. completion: Oct 2025Updated: Aug 2025

Active, Not RecruitingGene Therapy

NCT02633943

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

Sponsor:Genetix Biotherapeutics Inc.

United StatesStarted: Jan 2014Est. completion: Nov 2035Updated: 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 19, 2026 at 3:26 AM

Beta-Thalassemia Hbb/lcrb — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

50 Trials

Open Trials40

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

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 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 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

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

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 Trials10

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

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

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

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

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

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

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

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