Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study
Phase 1
- Conditions
- Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome)
- Registration Number
- JPRN-UMIN000009758
- Lead Sponsor
- Tokai University School of Medicine
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 12
Inclusion Criteria
Not provided
Exclusion Criteria
1. Down syndrome 2. HIV-positivity
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The incidence of severe RRT (Grade III/IV) on day 28 after HSCT with Treosulfan as a conditioning regimen.
- Secondary Outcome Measures
Name Time Method Regimen-related toxicity, engraftment rate, survival rate at 28 days posttransplant, survival rate at 100 days posttransplant, chimeric study, GVHD, hepatic SOS, event-free survival and overall survival at 1 year posttransplant.