Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Active, not recruitingOBSERVATIONAL
Enrollment

15

Participants

Timeline

Start Date

April 23, 2021

Primary Completion Date

March 31, 2024

Study Completion Date

March 31, 2024

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.

Trial Locations (1)

20132

Ospedale San Raffaele, Milan

All Listed Sponsors
lead

Fondazione Telethon

OTHER