ong-term follow-up of the WAS gene therapy study

Phase 1

• Conditions: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiencycaused by mutations in a single gene ,the Wiskott-Aldrich SyndromeProtein (WASP). WAS is characterised by micro-thrombocytopenia,recurrent infections,eczema and associated with a high incidence ofauto-immunity and of lymphoid malignancies. Over 150 uniquemutations in the WAS gene have been identified.Loss-of-functionmutations in this gene have widespread consequences on hematopoieticlineages.; MedDRA version: 19.1Level: PTClassification code 10047992Term: Wiskott-Aldrich syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-000274-20-GB
• Lead Sponsor: Genethon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03-12
• Last Update Posted: 31 January 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 10

Inclusion Criteria

1.Patients enrolled in the phase I/II 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 (GTG 002.07 and GTG 003. 08)
2.Parents, guardians or patient signed informed consent

Are the trial subjects under 18? yes
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1.Parents, guardians, patients unwilling to return for follow up during the 3 years long term period

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified