Ignite Creation Date:
2025-12-24 @ 4:55 PM
Ignite Modification Date:
2025-12-27 @ 7:57 PM
Study NCT ID:
NCT01899950
Status:
COMPLETED
Last Update Posted:
2016-01-06
First Post:
2013-07-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Sponsor:
Mayo Clinic
Organization:
Study Overview
Official Title:
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Status:
COMPLETED
Status Verified Date:
2016-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
NPC
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 with cerebellar ataxia (an inability to coordinate balance, gait, extremity and eye movements), dysarthria (difficulty speaking), seizures, vertical gaze palsy (ability to move eyes in the same direction) motor impairment, dysphagia (trouble swallowing), psychotic episodes, and progressive dementia. There is no curative treatment for NPC and it is a lethal disorder. The purpose of this protocol is to obtain both baseline and rate of progression data on a clinical and biochemical markers that may later be used as outcome measures in a clinical trial. Specifically, this study will examine and characterize the longitudinal progression of neurocognitive symptoms of NPC with the goal of identifying early markers of disease progression that may be utilized in later trials to evaluate treatment efficacy.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| U54NS065768 | NIH | None | https://reporter.nih.gov/quic… | View |
| LDN6715 | None | None | View |