Clinical Trial

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

Study acronym: MPSIIIC
Recruiting
View on ClinicalTrials.gov →
Summary
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
Protocol Amendment History 6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2023-04-20; most recent amendment 2026-05-07.
Status change: Not Yet Recruiting → Recruiting 2024-11-11
Trial Details
NCT Number NCT05825131
Lead Sponsor Phoenix Nest
Collaborators: Vaincres Les Maladies Lysosomales, Association Sanfilippo Sud, Aparito Ltd.
Conditions Sanfilippo Syndrome Type C
Enrollment 30 participants
Start Date 2024-11-10
Primary Completion 2028-07-30 (estimated)
Study Completion 2028-12-01 (estimated)
Updated on ClinicalTrials.gov 2026-05-12