Ignite Creation Date:
2024-05-06 @ 12:00 PM
Last Modification Date:
2024-10-26 @ 12:53 PM
Study NCT ID:
NCT03655678
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-13
First Post:
2018-08-29
Brief Title:
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia
Sponsor:
Vertex Pharmaceuticals Incorporated
Organization:
Vertex Pharmaceuticals Incorporated
Study Overview
Official Title:
A Phase 123 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34 Human Hematopoietic Stem and Progenitor Cells hHSPCs in Subjects With Transfusion-Dependent β-Thalassemia
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-09
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:
This is a single-arm open-label multi-site single-dose Phase 123 study in subjects with transfusion-dependent β-thalassemia TDT The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34 Human Hematopoietic Stem and Progenitor Cells hHSPCs using CTX001
Detailed Description:
None
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?:
True
Is an FDA AA801 Violation?:
None