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

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

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

A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894•Editas Medicine, Inc.

View on ClinicalTrials.gov

Summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Detailed Description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive

Eligibility

Age

18 - 35 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Diagnosis of Transfusion Dependent B-Thalassemia as defined by:
•Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and
•History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent
•Clinically stable and eligible to undergo autologous HSCT
•Karnofsky Performance Status ≥ 70

Exclusion Criteria

•Available 10/10 human leukocyte antigen (HLA)-matched related donor
•Prior HSCT or contraindications to autologous HSCT
•Participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records
•Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records
•Prior receipt of gene therapy
•Inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/L or a platelet count \< 100 x 10\^9/L (without hypersplenism), per investigator judgement
•Inadequate organ function
•Advanced liver disease
•Any prior or current malignancy, or immunodeficiency disorder,
•Immediate family member with a known or suspected Familial Cancer Syndrome
+1 more criteria

Locations (8)

University of California San Francisco

Oakland, California, United States

University of Minnesota

Minneapolis, Minnesota, United States

Columbia University Medical Center - Department of Pediatrics

New York, New York, United States

Columbia University Medical Center

New York, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers

Nashville, Tennessee, United States

Princess Margaret Cancer Centre-University Health Network

Toronto, Ontario, Canada

Key Dates

Start Date

April 29, 2022

Primary Completion Date

September 1, 2025

Completion Date

December 1, 2025

Last Updated

April 2, 2025

Enrollment

ESTIMATED participants

Conditions

Transfusion Dependent Beta ThalassemiaHemoglobinopathiesThalassemia MajorThalassemia Intermedia

Interventions

EDIT-301

GENETIC

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

NCT03128996

Severe Sickle Cell DiseaseBone Marrow Failure Syndromes+4 more

View study

RECRUITINGPHASE2

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

NCT07292259

Transfusion Dependent Beta Thalassemia

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 2, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

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

Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

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

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

Inspiratory Muscle Training in Children With Beta Thalasemia

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