Beta Thalassemia — Clinical Trials

MONDO:001940243 active·20 recruitingLast updated: Mar 20, 2026, 12:51 PM

Trial Landscape

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

Recruiting

Active Trials

By Approach

Drug Treatment17

Biological Therapy8

Gene Therapy4

Procedure3

Medical Device1

16 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/Other29Industry9NIH5

Top Sponsors

Bristol-Myers Squibb

Editas Medicine, Inc.

Pharmacosmos A/S

Shanghai BDgene Co., Ltd.

Vertex Pharmaceuticals Incorporated

Trial Locations

United States16 trials

Italy8 trials

China5 trials

Canada4 trials

Germany3 trials

Egypt3 trials

United Kingdom2 trials

Pakistan2 trials

India1 trial

Denmark1 trial

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

43 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 IncorporatedOrphan Designation

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

NCT07292259

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

Sponsor:Pakistan Blood and Marrow Transplant (PBMT) Group

PakistanStarted: Jan 2024Est. completion: Dec 2025Updated: Dec 2025

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

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 1Recruiting

NCT05904093

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

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

United StatesStarted: Dec 2024Est. completion: May 2026Updated: Feb 2026

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

Recruiting

NCT05508932

Atrial Fibrillation in Beta-Thalassemia

Sponsor:University Hospital of Ferrara

ItalyStarted: Sep 2022Est. completion: Jul 2032Updated: Feb 2026

Recruiting

NCT03937817

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Sponsor:National Institute of Allergy and Infectious Diseases (NIAID)

United StatesStarted: Sep 2019Est. completion: Mar 2029Updated: Feb 2026

Not ApplicableRecruiting

NCT06931912

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

Sponsor:Institute of Hematology & Blood Diseases Hospital, China

ChinaStarted: Dec 2024Est. completion: Nov 2026Updated: Feb 2026

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 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.Orphan Designation

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

Phase 1Active, Not RecruitingGene Therapy

NCT01639690

ß-Thalassemia Major with Autologous CD34+ Hematopoietic Progenitor Cells Transduced with TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene

Sponsor:San Rocco Therapeutics

ItalyStarted: Jul 2012Est. completion: Jul 2025Updated: Oct 2024

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

NCT05675436

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

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

United StatesStarted: May 2024Est. completion: Oct 2027Updated: Mar 2026

Active, Not Recruiting

NCT06596642

A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

Sponsor:Bristol-Myers Squibb

TaiwanStarted: Aug 2024Est. completion: Dec 2026Updated: Jun 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 20, 2026 at 12:51 PM

Beta Thalassemia — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

43 Trials

Open Trials31

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)

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

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

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

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

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

Atrial Fibrillation in Beta-Thalassemia

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

Active Trials12

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

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)

ß-Thalassemia Major with Autologous CD34+ Hematopoietic Progenitor Cells Transduced with TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene

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

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan

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