Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Severe Combined Immunodeficiency Due to ADA Deficiency Clinical Trial

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

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency Due to ADA Deficiency

• NCT number: NCT04959890
• Study type: Observational
• Source: Fondazione Telethon
• Status: Active, not recruiting
• Start date: April 23, 2021
• Completion date: March 31, 2024