Gene Therapy for Wiskott-Aldrich Syndrome (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

NCT01347242

GTG002.07

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: 6 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 or of a 10/10 antigen HLA-matched unrelated donor or 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 antigen HLA-matched unrelated donor or cord blood
• contraindication to leukapheresis
• contraindication to bone marrow harvest
• contraindication to administration of conditioning medication
• HIV positive patient

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

study treatment

Experimental group

Description:

autologous CD34 positive cells transduced with a lentiviral vector containing the human WAS gene

Treatment:

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