Ignite Creation Date:
2024-05-06 @ 5:50 PM
Last Modification Date:
2024-10-26 @ 2:36 PM
Study NCT ID:
NCT05444894
Status:
RECRUITING
Last Update Posted:
2024-01-29
First Post:
2022-06-27
Brief Title:
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant HSCT in Participants With Transfusion-Dependent Beta Thalassemia TDT
Sponsor:
Editas Medicine Inc
Organization:
Editas Medicine Inc
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2024-01
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 safety tolerability and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
Detailed Description:
This is a Phase 12 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 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