Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

NCT04959890 | Unknown

• 1 other identifier: STRIM-005
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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

Keywords

ada-scid; retroviral vector; gene therapy

Outcome Measures

Primary Outcomes (2)

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

  Precision will be determined by the variability (%CV) of the abundance data.

  Retrospective sample analysis.

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

  Accuracy will be determined based on the difference between the mean retrieved abundance and the expected abundance of control DNA.

  Retrospective sample analysis.

Study Arms (1)

Subjects Previously Treated with Strimvelis (or GSK2696273) Gene Therapy

It is expected that this study will include approximately 70 eligible samples from approximately 15 subjects previously treated with gamma retroviral gene therapy (gRV-GT).

Genetic: Strimvelis

Interventions

Strimvelis GENETIC

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.

Subjects Previously Treated with Strimvelis (or GSK2696273) Gene Therapy

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study will use biological samples previously taken from subjects treated with Strimvelis gRV-GT.

You may qualify if:

• Subjects must have previously received treatment with gRV-GT, either during clinical development (clinical trials and early access programs) or in the post-marketing setting as the approved product (Strimvelis) or under hospital exemption, and for whom at least one biological sample is available that meets the following eligibility 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).

Sponsors & Collaborators

• Fondazione Telethon: lead

Study Sites (1)

Ospedale San Raffaele

Milan, 20132, Italy

Location

MeSH Terms

Conditions

Severe combined immunodeficiency due to adenosine deaminase deficiency

Study Officials

• Fondazione Telethon

  Fondazione Telethon

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2021
• First Posted: July 13, 2021
• Study Start: April 23, 2021
• Primary Completion: March 31, 2024
• Study Completion: March 31, 2024
• Last Updated: January 29, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)