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Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

NCT04959890 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2024-01-29

No results posted yet for this study

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.

Conditions

  • Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

GENETIC

Strimvelis

This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with gRV-GT. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal. Eligible samples will be shipped to a central laboratory and will undergo DNA extraction prior to S-EPTS/LM-PCR analysis. Each analysis run will include a control DNA sample and up to four subject samples. Each sample will be analysed in triplicate.

Sponsors & Collaborators

  • Fondazione Telethon

    lead OTHER

Principal Investigators

  • Fondazione Telethon · Fondazione Telethon

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-23
Primary Completion
2024-03-31
Completion
2024-03-31

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04959890 on ClinicalTrials.gov