ong-term follow-up of the WAS gene therapy study
Phase 1
Recruiting
- Conditions
- Treatment of Wiskott-Aldrich SyndromeMedDRA version: 20.0Level: PTClassification code: 10047992Term: Wiskott-Aldrich syndrome Class: 100000004850Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2024-512680-31-00
- Lead Sponsor
- Genethon
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 10
Inclusion Criteria
Patients enrolled in the phase I/II studies and treated by a single infusion of autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector for WAS conducted in France and United Kingdom (GTG002.07 and GTG003. 08), Parents, guardians or patient signed informed consent.
Exclusion Criteria
Parents, guardians, patients unwilling to return for the follow up study period.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method