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Observational Study for Sanfilippo Syndrome Type C (MPS IIIC)

This is an observational study for people with Sanfilippo Syndrome Type C (MPS IIIC), a rare genetic disorder. It's called a "natural history" study because it aims to understand how the disease progresses over time without testing any new medications. You or your loved one would participate remotely from home for two years. Every six months, you would use a smartphone app to record videos of daily activities and complete questionnaires about language and speech. This helps researchers understand how MPS IIIC affects daily living, language, and speech. The study is looking for about 35 participants between 1 and 25 years old who have a confirmed diagnosis of MPS IIIC through enzyme and genetic tests.

Study design
This is a remote observational study aiming to enroll about 35 participants with MPS IIIC to understand the disease's natural progression.
What's involved
You would participate remotely for two years, submitting videos and completing questionnaires every six months using a smartphone app.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to two years, with assessments every six months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07712003

Natural History to Assess Disease in Patients With MPS IIIC

Not Yet Recruiting
Not specifiedAges 1–25Observational
Phoenix Nest
~35 participants
Updated 2026-07-17 on ClinicalTrials.gov

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Daily living functions captured by video
Measured over Baseline to up to 2 years with 6-month time points.
+4 more outcomes measured

Conditions

Sanfilippo Syndrome Type C
Mucopolysaccharidosis (MPS) IIIC

Explore related trials

Mucopolysaccharidosis Type IIICHEPARAN-ALPHA-GLUCOSAMINIDE N-ACETYLTRANSFERASE

Where it's being run

1 sites across 1 states
Texas1

Who to contact

Holly Lawrence
[email protected]
2144044239
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

1\. ≥1 year and ≤ 25 years of age 2. Confirmed diagnosis of MPS IIIC by all of the following:

What this trial measures

  • Daily living functions captured by videoBaseline to up to 2 years with 6-month time points.

    Ability to perform activities of daily living.

  • Expressive and receptive language assessed by standard questionnairesBaseline to up to 2 years with 6-month time points.

    Ability to understand, comprehend and respond to communication.

  • Speech as assessed by standard questionnairesBaseline to up to 2 years; with 6-month time points.

    Analysis of speech quality.

  • Behavior as assessed by ability to perform daily functions as captured by videoBaseline to up to 2 years with 6-month time points.

    Quality of behavior as defined by ability to perform defined functions

  • Mobility as assessed by video and standard questionnairesBaseline up to 2 years with 6-month time points.

    Assessment of fine and gross motor abilities