Natural History to Assess Disease in Patients With MPS IIIC

Conditions

• Mucopolysaccharidosis (MPS) IIIC
• Sanfilippo Syndrome Type C

Eligibility Criteria

Sex

ALL

Age

1 Year - 25 Years

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

First listed

Jul 17, 2026

Start date

Nov 1, 2026

Status verified

Jul 2026

Primary completion

Nov 1, 2028

Completion

Feb 1, 2029

Study Design

Enrollment

35 participants (estimated)

Primary Outcome Measure

Daily living functions captured by video [ Time Frame: Baseline to up to 2 years with 6-month time points. ]

Central Contacts

• Holly Lawrence
  2144044239
  [email protected]
• Alyssa Boudreau
  [email protected]

Locations (1)

Find similar trials in Dallas, TX

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