Ignite Creation Date:
2025-12-24 @ 10:17 PM
Ignite Modification Date:
2025-12-24 @ 10:17 PM
Study NCT ID:
NCT04205435
Status:
TERMINATED
Last Update Posted:
2022-10-13
First Post:
2019-12-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
β-globin Restored Autologous HSC in β-thalassemia Major Patients
Sponsor:
Bioray Laboratories
Organization:
Study Overview
Official Title:
a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
Status:
TERMINATED
Status Verified Date:
2021-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Sponsor decision
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 center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
Detailed Description:
β-globin restored autologous hematopoietic stem cells will be manufactured using CRISPR/Cas9 gene editing system. Subject participation for this study will be 1 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
Study Oversight
Has Oversight DMC:
False
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?: