Ignite Creation Date:
2024-05-06 @ 5:54 PM
Last Modification Date:
2024-10-26 @ 2:38 PM
Study NCT ID:
NCT05477563
Status:
RECRUITING
Last Update Posted:
2024-05-16
First Post:
2022-07-26
Brief Title:
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Sponsor:
Vertex Pharmaceuticals Incorporated
Organization:
Vertex Pharmaceuticals Incorporated
Study Overview
Official Title:
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34 Human Hematopoietic Stem and Progenitor Cells CTX001 in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
Status:
RECRUITING
Status Verified Date:
2024-03
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-dose open-label study in participants with transfusion-dependent β-thalassemia TDT or severe sickle cell disease SCD 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?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2021-006390-37 | EUDRACT_NUMBER | None | None |