Gene therapy for Wiskott-Aldrich Syndrome (WAS)
- Conditions
- Wiskott-Aldrich SyndromeHaematological Disorders
- Registration Number
- ISRCTN30718752
- Lead Sponsor
- Genethon (France)
- Brief Summary
2015 results in: https://www.ncbi.nlm.nih.gov/pubmed/25898053 (added 11/04/2019)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Male
- Target Recruitment
- 5
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. Unless desease severity indicates that one cannot wait for 3 months (score 5; refractory thrombocytopenia with platelets < 5000 with bleeding or severe autoimmunity)
5. Lack of HLA-genotypically identical bone marrow after 3 month search
6. Lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search
7. Lack of a HLA-matched cord blood after 3 month search
8. Parental, guardian, patient signed informed consent/assessment
9. Willing to return for follow-up during the 2 year study and lifelong for off study review
10. Only for patients who have received previous allogenic haematopoietic stem cell transplant
10.1. Failed allogenic haematopoietic stem cell transplant
10.2. Contraindication to repeat allogenic transplantation
1. Patient with HLA-genotypically identical bone marrow
2. Patient with 10/10 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood
3. Contraindication to leukapheresis
3.1. Anaemia (Hb < 8g/dl)
3.2. Severe vascularitis
3.3. Refractory thrompopenia
3.3.1. Contraindication to bone marrow harvest
3.3.2. Contraindication to administration of conditioning medication
4. Human immunodeficiency virus (HIV) seropositive patient
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method