A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
- Conditions
- Wiskott-Aldrich syndrome
- Registration Number
- JPRN-UMIN000030806
- Lead Sponsor
- ational Center for Child Heath and Development
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up continuing
- Sex
- Male
- Target Recruitment
- 3
Not provided
Patients who meet any of the following criteria will be excluded. 1. Patients positive for HIV infection 2. Patients affected by neoplasia 3. Patients with cytogenetic alterations typical of MDS/AML 4. Patients with end-organ function or any other severe disease, which, in the judgement of the investigator, would make the patients inappropriate for entry into this study 5. Patients who underwent an allogeneic hematopoietic stem cell transplantation in the previous 6 months 6. Patients who underwent an allogeneic hematopoietic stem cell transplantation with evidence of residual donor cells 7. Patients who have the possibility of severe allergic reactions, to rituximab and the products derived from cow, pig, sheep and mouse. 8. Patients who do not agree with a contraception during the trial. 9. Patients who are considered inappropriate, in the judgement of the investigator, due to any other reasons.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method