A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C
Not Applicable
- Conditions
- iemann-Pick disease type C
- Registration Number
- JPRN-UMIN000023517
- Lead Sponsor
- ara Medical University
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 1
Inclusion Criteria
Not provided
Exclusion Criteria
1)CNS or systemic infection 2)Without neurological involvement
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method PC-ADL score after 12th administration of HPBCD
- Secondary Outcome Measures
Name Time Method