Ignite Creation Date:
2024-05-06 @ 4:14 PM
Last Modification Date:
2024-10-26 @ 2:06 PM
Study NCT ID:
NCT04925206
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-02-17
First Post:
2021-06-04
Brief Title:
A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia
Sponsor:
EdiGene GuangZhou Inc
Organization:
EdiGene GuangZhou Inc
Study Overview
Official Title:
A Multicenter Open Label Phase 1 Study to Evaluate 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-Thalassaemia
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-02
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:
ET-01
Brief Summary:
This is a single-arm open label multi-center single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34 Human Hematopoietic Stem and Progenitor Cells hHSPCs using ET-01
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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