UnknownNot Applicable

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Italy15 participantsStarted 2021-04-23

Plain-language summary

This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.

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

. Peripheral blood, bone marrow, or DNA extracted from either source.
. Taken at least 6 months after gRV-GT.
. Stored at -20oC or below since the time of sampling.
. Likely to provide at least 1.5 μg of DNA (following extraction by the central laboratory).

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

To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA

Timeframe: Retrospective sample analysis.

To assess the accuracy of S-EPTS/LM-PCR methodology for RIS using control insertion site DNA

Timeframe: Retrospective sample analysis.

Trial details

NCT IDNCT04959890

SponsorFondazione Telethon

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2024-03-31

View on ClinicalTrials.gov More Severe Combined Immunodeficiency Due to ADA Deficiency trials

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

. Peripheral blood, bone marrow, or DNA extracted from either source.
. Taken at least 6 months after gRV-GT.
. Stored at -20oC or below since the time of sampling.
. Likely to provide at least 1.5 μg of DNA (following extraction by the central laboratory).