Viewing Study NCT04853576



Ignite Creation Date: 2024-05-06 @ 4:02 PM
Last Modification Date: 2024-10-26 @ 2:02 PM
Study NCT ID: NCT04853576
Status: ACTIVE_NOT_RECRUITING
Last Update Posted: 2024-07-12
First Post: 2021-04-16

Brief Title: A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease RUBY
Sponsor: Editas Medicine Inc
Organization: Editas Medicine Inc

Study Overview

Official Title: A Phase 12 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
Status: ACTIVE_NOT_RECRUITING
Status Verified Date: 2024-10
Last Known Status: None
Delayed Posting: No
If Stopped, Why?: Not Stopped
Has Expanded Access: False
If Expanded Access, NCT#: N/A
Has Expanded Access, NCT# Status: N/A
Acronym: None
Brief 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
Detailed Description: This is a Phase 12 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 Oversight

Has Oversight DMC: None
Is a FDA Regulated Drug?: True
Is a FDA Regulated Device?: False
Is an Unapproved Device?: None
Is a PPSD?: None
Is a US Export?: None
Is an FDA AA801 Violation?: None