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

Hemoglobinopathies Clinical Trial

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)

Clinical Trial 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

Clinical Trial 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 Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05444894
Study type Interventional
Source Editas Medicine, Inc.
Contact Editas Medicine Clinical Trial Team
Phone 617-401-9007
Email Patients@editasmed.com
Status Recruiting
Phase Phase 1/Phase 2
Start date April 29, 2022
Completion date December 2025
View Details
See also
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