Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC)
Not Applicable
- Conditions
- iemann-Pick disease type C
- Registration Number
- JPRN-UMIN000020032
- Lead Sponsor
- Jichi Medical University, Saitama Medical Center
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up continuing
- Sex
- All
- Target Recruitment
- 2
Inclusion Criteria
Not provided
Exclusion Criteria
1) Infantile and child NPC 2) CNS or systemic infection 3) Without neurological involvement
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method PC-ADL score one year after starting intervention
- Secondary Outcome Measures
Name Time Method hearing loss, pulmonary lesion, serum oxysterol, urinary bile acids