Gene Therapy for WAS

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-05-31

Study Completion Date

2017-01-09

Brief Summary

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

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Detailed Description

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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.

Conditions

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Wiskott-Aldrich Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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

transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene

Intervention Type GENETIC

Eligibility Criteria

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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

Eligible Sex

MALE

Accepts Healthy Volunteers

No