Ignite Creation Date:
2024-05-06 @ 10:39 AM
Last Modification Date:
2024-10-26 @ 12:33 PM
Study NCT ID:
NCT03311074
Status:
WITHDRAWN
Last Update Posted:
2024-01-29
First Post:
2017-10-11
Brief Title:
Retroviral Insertion Site Methodology Study
Sponsor:
Fondazione Telethon
Organization:
Fondazione Telethon
Study Overview
Official Title:
Methodology Study to Investigate the Utility of Retroviral Insertion Site Analysis in Samples From Subjects Treated With Strimvelis Gene Therapy
Status:
WITHDRAWN
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Replaced with new observational study design
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Adenosine deaminase ADA is an enzyme involved in the development and functioning of the immune system Deficiency of ADA results in severe combined immunodeficiency SCID a fatal inherited immune disorder Strimvelis is a gene therapy that aims to insert ADA function into blood cells and halt or reverse the conditions caused by decreased ADA enzyme levels such as impaired immune function It is important to consider long term follow-up of patients who have received Strimvelis including evaluation of the risk of insertion near certain genes that may lead to unexpected activation of those genes oncogenesis The objective of this study is to evaluate the use of a new technique to identify where Strimvelis has become inserted in the genetic sequence and potential implications for patient care This new technique is known as sonication linker mediated polymerase chain reaction SLiM-PCR for retroviral insertion site RIS analysis The study will recruit at least 15 pediatric or adult patients with ADA-SCID who have been treated with Strimvelis either in previous clinical trials or as a registered product Recruitment for the study may remain open for up to 2 years even if 15 subjects are recruited sooner Study participation will last for up to 5 years A total of 5 blood samples will be collected from each subject at approximately annual intervals
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