Inherited Hemoglobinopathy — Clinical Trials

MONDO:001905028 active·14 recruitingLast updated: Mar 16, 2026, 4:24 AM

Trial Landscape

Inherited Hemoglobinopathy has an active clinical trial landscape, driven by drug treatment and biological therapy.

Recruiting

Active Trials

By Approach

Drug Treatment12

Biological Therapy12

Gene Therapy4

Medical Device2

Procedure2

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

By Phase

Early Phase 1

Phase 1

Phase 2

Phase 3

7 trials are observational or have no phase designation.

Sponsor Types

Academic/Other17Industry10NIH1

Top Sponsors

Beam Therapeutics Inc.

Bellicum Pharmaceuticals

Editas Medicine, Inc.

ScinnoHub Pharmaceutical Co., Ltd.

Vertex Pharmaceuticals Incorporated

Trial Locations

United States23 trials

Canada6 trials

Italy5 trials

Germany4 trials

China3 trials

United Kingdom3 trials

Belgium2 trials

Pakistan2 trials

Angola1 trial

Argentina1 trial

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

28 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 Incorporated

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

Phase 2Recruiting

NCT04644016

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Sponsor:Memorial Sloan Kettering Cancer Center

United StatesStarted: Nov 2020Est. completion: Dec 2026Updated: Mar 2026

Phase 2Recruiting

NCT06872333

Allo HSCT for High Risk Hemoglobinopathies

Sponsor:Masonic Cancer Center, University of Minnesota

United StatesStarted: Nov 2024Est. completion: Jun 2032Updated: Mar 2026

Phase 2Recruiting

NCT04356469

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Sponsor:Johns Hopkins All Children's Hospital

United StatesStarted: Jul 2020Est. completion: Jun 2026Updated: Jun 2025

Phase 1Recruiting

NCT00920972

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

Sponsor:Washington University School of Medicine

United StatesStarted: Dec 2001Est. completion: Dec 2031Updated: Mar 2026

Phase 1Recruiting

NCT03128996

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

Sponsor:Washington University School of Medicine

United StatesStarted: Mar 2017Est. completion: Apr 2033Updated: Jan 2026

Phase 1Recruiting

NCT07087262

A Phase I Study of SNH-119014 in Healthy Volunteers

Sponsor:ScinnoHub Pharmaceutical Co., Ltd.

ChinaStarted: Jun 2025Est. completion: Mar 2026Updated: Jul 2025

Phase 1Recruiting

NCT06839456

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Sponsor:Children's Hospital of Philadelphia

United StatesStarted: Mar 2025Est. completion: Mar 2032Updated: Mar 2025

Early Phase 1Recruiting

NCT06313398

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

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

United StatesStarted: May 2024Est. completion: Jun 2029Updated: Feb 2026

Early Phase 1Recruiting

NCT06107400

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Sponsor:The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

ChinaStarted: Oct 2023Est. completion: Oct 2026Updated: Jun 2024

Last updated over 18 months ago

Phase 3Active, Not Recruiting

NCT05329649

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sponsor:Vertex Pharmaceuticals Incorporated

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

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

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

NCT01050855

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

Sponsor:Children's Hospital of Philadelphia

United StatesStarted: Jan 2008Est. completion: Dec 2026Updated: Jul 2024

Last updated over 18 months ago

Phase 1Active, Not Recruiting

NCT03249831

A Blood Stem Cell Transplant for Sickle Cell Disease

Sponsor:City of Hope Medical Center

United StatesStarted: Jan 2019Est. completion: Jan 2027Updated: Mar 2026

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

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

Phase 1Active, Not Recruiting

NCT04853576

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

Sponsor:Editas Medicine, Inc.

United StatesStarted: May 2021Est. completion: Aug 2025Updated: Jan 2025

Phase 1Active, Not Recruiting

NCT03301168

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

Sponsor:Bellicum Pharmaceuticals

United StatesStarted: Apr 2014Est. completion: May 2034Updated: Jul 2022

Last updated over 18 months ago

Not ApplicableActive, Not Recruiting

NCT05236764

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

Sponsor:Baylor College of Medicine

United StatesStarted: Dec 2023Est. completion: May 2026Updated: Oct 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 16, 2026 at 4:24 AM

Inherited Hemoglobinopathy — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

28 Trials

Open Trials18

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

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Allo HSCT for High Risk Hemoglobinopathies

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

A Phase I Study of SNH-119014 in Healthy Volunteers

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Active Trials10

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

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

Long Term Beta Thalassemia Treatment: Findings From The Extension Period

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

A Blood Stem Cell Transplant for Sickle Cell Disease

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

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

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion