Gene Therapy for WAS

Genethon

Status and phase

Completed

Phase 2

Phase 1

Conditions

Wiskott-Aldrich Syndrome

Treatments

Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

Study type

Interventional

Funder types

Other

Identifiers

NCT01347346

GTG003.08

2009-011152-22 (EudraCT Number)

Details and patient eligibility

About

This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.

Full description

This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene.

Enrollment

5 patients

Sex

Male

Volunteers

No Healthy Volunteers

Inclusion criteria

males of all ages
severe WAS (clinical score 3-5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
molecular confirmation by WAS gene DNA sequencing
lack of HLA-genotypically identical bone marrow after 3 month search
lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search
lack of a HLA-matched cord blood after 3 month search
parental, guardian, patient signed informed consent/assent
willing to return for follow-up
only for patients who have received previous allogenic hematopoietic stem cell transplant:
failed allogenic hematopoietic stem cell transplant
contraindication to repeat transplantation

Exclusion criteria

patient with HLA-genotypically identical bone marrow
patient with 10/10 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood
contraindication to leukapheresis
contraindication to bone marrow harvest
contraindication to administration of conditioning medication
HIV positive patient