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

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Brief Title

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

Official Title

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)

Brief 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

Study Phase

Phase 1/Phase 2

Study Type


Primary Outcome

Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)

Secondary Outcome

 Kinetics of HSPC engraftment


Transfusion Dependent Beta Thalassemia



Study Arms / Comparison Groups

Description:  EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

April 29, 2022

Completion Date

December 2025

Primary Completion Date

September 2025

Eligibility Criteria

        Key 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

        Key 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

          -  Clinically significant and active bacterial, viral, fungal, or parasitic infection




18 Years - 35 Years

Accepts Healthy Volunteers



, 617-401-9007, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Editas Medicine, Inc.

Study Sponsor

, , 

Verification Date

October 2022