Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (WASFUP)

Genethon

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Wiskott-Aldrich Syndrome

Treatments: Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector

Study type: Interventional

Funder types: Other

Identifiers

NCT02333760

GNT-WAS-03

Details and patient eligibility

About

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.

Enrollment: 10 estimated patients

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).
• Parents, guardians or patient signed informed consent, guardians or patient signed informed consent

Exclusion criteria

• Parents, guardians, patients unwilling to return for the follow up study period.

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)