Gene therapy for Wiskott-Aldrich Syndrome (WAS)

Phase 1

Completed

• Conditions: Wiskott-Aldrich Syndrome; Haematological Disorders; Wiskott-Aldrich syndrome

• Registration Number: ISRCTN46087965
• Lead Sponsor: Genethon (France)

Brief Summary

2017 results in: https://www.ncbi.nlm.nih.gov/pubmed/28716862

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-05-20
• Study Completion: 2013-12-31
• Last Update Posted: 30 April 2018

Recruitment & Eligibility

• Status: Completed
• Sex: Male
• Target Recruitment: 5

Inclusion Criteria

1. Males of all ages
2. Severe WAS (clinical score 3 ? 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
3. Molecular confirmation by WAS gene DNA sequencing
4. Lack of HLA-genotypically identical bone marrow or of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search
5. Parental, guardian, patient signed informed consent/assessment
6. Willing to return for follow-up during the 2 year study and the 3 year long-term off study review
7. Only for patients who have received previous allogenic haematopoietic stem cell transplant:
7.1. Failed allogenic haematopoietic stem cell transplant
7.2. Contraindication to repeat allogeneic transplantation for example severe graft versus host disease

Exclusion Criteria

1. Patient with HLA-genotypically identical bone marrow
2. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood
3. Contraindication to leukapheresis
3.1. Anaemia (Hb < 8g/dl)
3.2. Cardiovascular instability
3.3. Severe coagulopathy
3.3.1. Contraindication to bone marrow harvest
3.3.2. Contraindication to administration of conditioning medication
3. Human immunodeficiency virus (HIV) positive patient

Study & Design

• Study Type: Interventional
• Study Design: Not specified