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

Interventional


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

Condition

Transfusion Dependent Beta Thalassemia

Intervention

EDIT-301

Study Arms / Comparison Groups

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

Publications

* 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

Genetic

Estimated Enrollment

6

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
      

Gender

All

Ages

18 Years - 35 Years

Accepts Healthy Volunteers

No

Contacts

, 617-401-9007, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT05444894

Organization ID

EM-301-BThal-001


Responsible Party

Sponsor

Study Sponsor

Editas Medicine, Inc.


Study Sponsor

, , 


Verification Date

October 2022