Ignite Creation Date:
2024-05-06 @ 1:50 PM
Last Modification Date:
2024-10-26 @ 1:21 PM
Study NCT ID:
NCT04140539
Status:
WITHDRAWN
Last Update Posted:
2022-08-03
First Post:
2019-10-24
Brief Title:
A Clinical Study to Enable Process Validation of Commercial Grade OTL-101
Sponsor:
University of California Los Angeles
Organization:
University of California Los Angeles
Study Overview
Official Title:
A Single Arm Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy OTL-101 in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency ADA-SCID
Status:
WITHDRAWN
Status Verified Date:
2022-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Recruitment on hold for business reasons Study will not be performed
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process
Detailed Description:
The safety and efficacy of OTL-101 for the treatment of patients with ADA- SCID have been established in previous clinical trials The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process in order to facilitate collection of data necessary for final manufacturing
Assessments will focus on monitoring safety and engraftment through the evaluation of parameters describing immunological recovery ADA enzyme activity and persistence of gene marking VCN at 6 months and 12 months After completion of 12 months of follow-up on the current study protocol subjects will be enrolled in an observational long-term follow-up study in order to monitor the long-term safety of treatment with OTL-101
Assessments will focus on monitoring safety and engraftment through the evaluation of parameters describing immunological recovery ADA enzyme activity and persistence of gene marking VCN at 6 months and 12 months After completion of 12 months of follow-up on the current study protocol subjects will be enrolled in an observational long-term follow-up study in order to monitor the long-term safety of treatment with OTL-101
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