Ignite Creation Date:
2024-05-06 @ 11:15 AM
Last Modification Date:
2024-10-26 @ 12:43 PM
Study NCT ID:
NCT03478670
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-01-29
First Post:
2018-03-23
Brief Title:
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency ADA-SCID
Sponsor:
Fondazione Telethon
Organization:
Fondazione Telethon
Study Overview
Official Title:
Adenosine Deaminase Severe Combined Immunodeficiency ADA-SCID Registry for Patients Treated With Strimvelis Previously GSK2696273 Gene Therapy Long-Term Prospective Non-Interventional Follow-up of Safety and Effectiveness
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-01
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:
Adenosine deaminase ADA enzyme deficiency results in severe combined immunodeficiency SCID a fatal autosomal recessive inherited immune disorder Strimvelis or GSK2696273 is a gene therapy intended for patients with ADA-SCID and for whom no suitable human leukocyte antigen HLA matched related stem cell donor is available This therapy aims to restore ADA function in hematopoietic cell lineages and in doing so prevents the pathology caused by purine metabolites ie impaired immune function This registry evaluates the long term safety and effectiveness outcomes of subjects who have received Strimvelis or GSK2696273
Detailed Description:
This is a prospective non-interventional follow-up registry of patients with ADA-SCID treated with Strimvelis The registry does not have a comparator group and the product will have been given on a single occasion prior to entering this registry Safety and effectiveness will be assessed for a target number of 50 patients who will have received Strimvelis or GSK2696273 comprising patients treated prior to marketing authorisation ie clinical studies and compassionate use programs and those treated after marketing authorisation including within compassionate use and early access programs The registry will close to enrolment when 50 patients have been enrolled but will not close completely until the 50th patient finishes their 15 year follow-up
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