Status:
COMPLETED
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Lead Sponsor:
Mayo Clinic
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Center for Advancing Translational Sciences (NCATS)
Conditions:
Niemann-Pick Disease, Type C
Eligibility:
All Genders
2-99 years
Brief Summary
Niemann-Pick Disease, Type C (NPC) is a rare neurodegenerative disorder with a wide clinical spectrum and variable age of onset. Classically, children with NPC demonstrate neurological dysfunction wit...
Eligibility Criteria
Inclusion
- All individuals between the ages of 2 years and 99 with an established diagnosis of Niemann-Pick Disease, Type C (biochemical or molecular) will be considered for this study. Patients with both Niemann-Pick Disease, Type C1 (NPC1) and Niemann-Pick Disease, Type C2 (NPC2) mutations are eligible to participate.
Exclusion
- Participants with at least one of the following will not be eligible for this study:
- Individuals that cannot travel because of their medical condition or are too ill to be cared for at home,
- Individuals with stage 4 disease (non-ambulant with vegetative disturbances)
- Individuals will be excluded if English is not their primary language
Key Trial Info
Start Date :
March 1 2011
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 1 2014
Estimated Enrollment :
70 Patients enrolled
Trial Details
Trial ID
NCT01899950
Start Date
March 1 2011
End Date
September 1 2014
Last Update
January 6 2016
Active Locations (1)
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1
Mayo Clinic
Rochester, Minnesota, United States, 55905