Ignite Creation Date:
2024-05-06 @ 8:13 PM
Last Modification Date:
2024-10-26 @ 3:23 PM
Study NCT ID:
NCT06300723
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-05-21
First Post:
2024-03-03
Brief Title:
Clinical Study of BRL-101 in Severe SCD
Sponsor:
Bioray Laboratories
Organization:
Bioray Laboratories
Study Overview
Official Title:
Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of CRISPRCas9-edited Autologous CD34 Hematopoietic StemProgenitor Cells BRL-101 in the Treatment of Severe Sickle Cell Disease
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-05
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 center non-randomized open label single-dose study in subjects with 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 BRL-101
Detailed Description:
This clinical trial is a single-arm single-dose single center open-label study without dose escalation The primary objective is to explore the safety of the study drug in SCD Myeloablative conditioning and administration for the remaining subjects can only be started after the first subject completes dosing and safety observation and assessment
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