Smartphone videos could unlock secrets of rare brain disease
NCT ID NCT07712003
First seen Jul 17, 2026 · Last updated Jul 17, 2026
Summary
This study tracks how Sanfilippo syndrome type C, a rare genetic disorder that affects the brain, progresses over time. Caregivers of children and young adults aged 1 to 25 will record videos of daily activities and answer questionnaires using a smartphone app every six months for two years. No experimental treatment is given; the goal is to better understand the disease's natural course and develop tools to measure changes in abilities like speech, movement, and behavior.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study could provide better ways to measure disease progression in Sanfilippo syndrome type C, helping future trials test treatments more effectively.
- What could go wrong
- This is an observational study with no treatment being tested, so it will not directly improve patient outcomes. The remote video approach may have limitations in capturing all aspects of the disease.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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UT Southwestern Children's Medical Center
Dallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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