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Natural History to Assess Disease in Patients With MPS IIIC

NCT07712003 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 35

Last updated 2026-07-17

No results posted yet for this study

Summary

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.

Conditions

  • Sanfilippo Syndrome Type C
  • Mucopolysaccharidosis (MPS) IIIC

Sponsors & Collaborators

  • National Institutes of Health (NIH)

    collaborator NIH

  • Phoenix Nest

    lead INDUSTRY

Eligibility

Min Age
1 Year
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-11-01
Primary Completion
2028-11-01
Completion
2029-02-01

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07712003 on ClinicalTrials.gov