Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

Genethon6 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Wiskott-Aldrich Syndrome

Interventions

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

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

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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

Locations (2)

Great Ormond Street Hospital

London, United Kingdom

Royal Free Hospital

London, United Kingdom

Outcomes

Primary Outcomes

Improvement in the eczema status

Improvement in the eczema status as compared with the baseline status at study entry on clinical evaluation

Time frame: 2 years

Reduction in the frequency and severity of infection episodes

Reduction in the frequency and severity of infection episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry

Time frame: 2 years

Reduction in the frequency and severity of bruising and bleeding episodes

Reduction in the frequency and severity of bruising and bleeding episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry

Time frame: 2 years

Reduction in the frequency and severity of autoimmune disorders

Reduction in the frequency and severity of autoimmune disorders as compared with the baseline status at study entry

Time frame: 2 years

Reduction in the number of disease related days of hospitalization

Reduction in the number of disease related days of hospitalization as compared with the patient's historical data collected over the 2 years prior to study entry

Time frame: 2 years

Secondary Outcomes

Occurrence and type of adverse events

Occurrence and type of adverse events reported during the course of the study

Time frame: 2 years

Change in medical conditions

Assessment of weight, vital signs, ECG and laboratory exams during the course of the study

Data from ClinicalTrials.gov

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