Status:
ENROLLING_BY_INVITATION
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Lead Sponsor:
Fondazione Telethon
Conditions:
Immunologic Deficiency Syndromes
Eligibility:
All Genders
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 inte...
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...
Eligibility Criteria
Inclusion
- Inclusion Criteria
- Patient with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program.
- Adult patients, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry.
- There are no formal exclusion criteria for participation as this registry will follow all patients who have received Strimvelis™ prior to enrollment, subject to informed consent.
Exclusion
Key Trial Info
Start Date :
March 28 2017
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 31 2037
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT03478670
Start Date
March 28 2017
End Date
May 31 2037
Last Update
January 29 2024
Active Locations (1)
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1
Ospedale San Raffaele
Milan, Lombardy, Italy, 20132