NCT04959890

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Methodology Study to Investigate the Utility of Retroviral Insertion Site Analysis in Samples From Subjects Treated With Strimvelis Gene Therapy.

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 15 (estimated)
• Sponsor: Fondazione Telethon · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with Strimvelis. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal. The objective of this methodology study is to evaluate the accuracy and precision of shearing extension primer tag selection ligation-mediated polymerase chain reaction (S-EPTS/LM-PCR) for RIS analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects previously treated with Strimvelis for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.

Conditions

• Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2021-04-23
• Primary completion: 2024-03-31
• Completion: 2024-03-31
• First posted: 2021-07-13
• Last updated: 2024-01-29

Locations

1 site across 1 country: Italy