Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Launched by FONDAZIONE TELETHON · Mar 23, 2018

Keywords

ClinConnect Summary

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 (i.e. clinical studies and compassionate use programs) and those treated after marketing authorisation (including within compassionate use and early access pr...

Gender

ALL

Eligibility criteria

• • Inclusion Criteria
• • 1. Patient with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program.
• • 2. 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.