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

Plain-language summary

This registry study provides long-term follow-up for patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID) who were treated with the Strimvelis gene therapy. It includes patients who previously received Strimvelis or the related gene therapy and provided consent. This is a non-phase follow-up study and is enrolling by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

Fondazione Telethon

Intervention

Strimvelis

Start date

5/5/2017

Estimated completion

12/31/2045

Countries

Italy

Related genes

Locations

• Milan, Italy

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria

1. Patients with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program or mPB-GT.
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™ or GSK2696273, or mPB-GT prior to enrollment, subject to informed consent.