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NCT01899950 · ClinicalTrials.gov registry record
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
A clinical trial, sponsored by Mayo Clinic.
- Completed
- Registry status
- 70
- Enrollment target
NCT01899950: Completed study, sponsored by Mayo Clinic.
NCT01899950 is a clinical trial that has completed, run by Mayo Clinic. The registered enrollment target is 70 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01899950 has completed, sponsored by Mayo Clinic.
- COMPLETED
- Registry status
- 70 participants
- Enrollment target
Study 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.
Primary Outcome
The composite score of neurocognitive measures will be calculated as follows: raw scores of neurocognitive tests will be transformed to Z-scores on normative data to allow comparison across measures administered at different ages. A score will be generated for each of the following domains: Intellectual Ability,Visual-Spatial Skills, Rote Verbal Memory, Narrative Verbal Memory, Visual-Spatial Construction, Nonverbal Working Memory, Language, Fine Motor, Attention, Executive Functioning, and Adap
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2011-03 |
| Est. Completion | 2014-09 |
What the finished NCT01899950 record still lists
NCT01899950 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 70 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT01899950 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01899950 about?
NCT01899950 is a clinical study titled "Longitudinal Study of Cognition With Niemann-Pick Disease, Type C". 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), dysarth...
What is the current status of trial NCT01899950?
This trial is currently completed. The enrollment target is 70 participants. The study started on 2011-03. Estimated completion is 2014-09.
Who is sponsoring clinical trial NCT01899950?
This trial is sponsored by Mayo Clinic, which has 2,844 total clinical trials registered on ClinicalTrials.gov.
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