By InvitationNot Applicable

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

Italy50 participantsStarted 2017-05-05

Plain-language 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 (i.e., impaired immune function). This registry evaluates the long term safety and effectiveness outcomes of subjects who have received Strimvelis and is conducted as a post approval safety study associated with EMA marketing authorisation of Strimvelis™. In this study will be also included patients for whom the gene therapy medicinal product has been prepared starting from mobilized peripheral blood (mPB)-derived CD34+ cells (mPB-GT).

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Patients with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program or mPB-GT.
. Adult patients, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Frequency of adverse events of special interest

Timeframe: Up to 15 years

Frequency of reported AEs and SAEs/ADRs

Timeframe: Up to 15 years

Actual values of laboratory blood test results (i.e. biochemistry, haematology) at each annual visits.

Timeframe: At each annual visit up to 15 years

Number (%) of subjects with fertility and positive pregnancy outcomes

Timeframe: Up to 15 years

The number (%) of subjects with an abnormal retroviral insertion site (RIS) analysis.

Timeframe: Up to 15 years.

Trial details

NCT IDNCT03478670

SponsorFondazione Telethon

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2045-12-31

View on ClinicalTrials.gov More Immunologic Deficiency Syndromes trials

Plain-language summary

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Patients with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program or mPB-GT.
. Adult patients, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry.

What they're measuring

Frequency of adverse events of special interest

Timeframe: Up to 15 years

Frequency of reported AEs and SAEs/ADRs

Timeframe: Up to 15 years

Actual values of laboratory blood test results (i.e. biochemistry, haematology) at each annual visits.

Timeframe: At each annual visit up to 15 years

Number (%) of subjects with fertility and positive pregnancy outcomes

Timeframe: Up to 15 years

The number (%) of subjects with an abnormal retroviral insertion site (RIS) analysis.

Timeframe: Up to 15 years.