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Natural History to Assess Disease in Patients With MPS IIIC

Study acronym: C-RARE
StatusNot Yet Recruiting
PhaseNot specified
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
This is a REMOTE prospective observational study of participants with MPS IIIC. No investigational medicinal product will be administered as part of this study. Patients functional abilities will be captured using video recordings taken by patients legal representative or caregiver using a dedicated clinical video application downloaded from the internet onto a smartphone. The video application is called C-RARE. Electronic observer-reported outcome (ObsRO) questionnaires will be completed by the legal representatives or caregivers via C-RARE. The study will last two years with videos and questionnaires taken every 6 months for a total of 5 time points. Data will then be reviewed and measured for functional change using a scoring scale designed for the use of this study.
Trial Details
NCT Number NCT07712003
Lead Sponsor Phoenix Nest
Collaborators: National Institutes of Health (NIH)
Conditions
(from sponsor)		 Sanfilippo Syndrome Type C, Mucopolysaccharidosis (MPS) IIIC
Related terms Mucopolysaccharidoses, Mucopolysaccharidosis III
Enrollment 35 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov