RecruitingNCT05825131

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

A Combination Retrospective and Prospective Natural History Study of Participants With Sanfilippo Syndrome Mucopolysaccharidosis Type IIIC (MPS IIIC)

Sponsor

Phoenix Nest

Enrollment

30 participants

Start Date

Nov 10, 2024

Study Type

OBSERVATIONAL

Conditions

Sanfilippo Syndrome Type C

Summary

This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.

Eligibility

Min Age: 12 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a new treatment for people with sanfilippo syndrome type c. The study is currently recruiting participants at 2 locations. People eligible for this study include aged 12 Months and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(2)

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Hospices Civils De Lyon

Bron, France

View Full Details on ClinicalTrials.gov

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NCT05825131