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

Sickle Cell Disease Clinical Trial

Official title:
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).

Clinical Trial Description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04853576
Study type Interventional
Source Editas Medicine, Inc.
Contact Editas Medicine's Clinical Trial Team
Phone 617-401-9007
Email Patients@editasmed.com
Status Recruiting
Phase Phase 1/Phase 2
Start date May 4, 2021
Completion date August 2025
View Details
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