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Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

Study acronym: MPSIIIC
StatusRecruiting
PhaseNot specified
Started
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Record History

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  1. Primary completion date: Jul 30, 2027 to Jul 30, 2028
    • Study completion date: Nov 1, 2027 to Dec 1, 2028
    • Site details revised at 1 of 2 sites
    v7
  2. Re-verified, no change to tracked fieldsv6
  3. Trial status: Not Yet Recruiting to Recruiting
    • Start date: Jun 10, 2024 to Nov 10, 2024
    • Site details revised at 2 of 2 sites
    v5
  4. Re-verified, no change to tracked fieldsv4
  5. Enrollment target: 15 to 30v3 Show 6 more changesShow less
    • Primary completion date: Oct 30, 2025 to Jul 30, 2027
    • Start date: Jun 10, 2023 to Jun 10, 2024
    • Study sites: 1 to 2
    • Study completion date: Mar 1, 2026 to Nov 1, 2027
    • Study description expanded, about 21 words
    • Study arms: 1 to 3
  6. Secondary endpoints: 4 to 7v2
  7. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
Trial Details
NCT Number NCT05825131
Lead Sponsor Phoenix Nest
Collaborators: Vaincres Les Maladies Lysosomales, Association Sanfilippo Sud, Aparito Ltd.
Conditions
(from sponsor)		 Sanfilippo Syndrome Type C
Related terms Mucopolysaccharidosis III
Enrollment 30 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov