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

SexALL

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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).

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