A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C
Not Applicable
Not yet recruiting
- 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
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms underlie cyclodextrin's efficacy in Niemann-Pick disease type C trials?
How does intrathecal cyclodextrin compare to oral miglustat in NPC treatment outcomes?
Which biomarkers correlate with response to HPBCD therapy in Niemann-Pick C patients?
What are the long-term safety concerns for cyclodextrin spinal administration in NPC treatment?
Are there synergistic effects when combining cyclodextrin with cholesterol-lowering agents for NPC therapy?