CBD trial aims to tame Sanfilippo's toughest symptoms
NCT ID NCT06333041
First seen Mar 10, 2026 · Last updated Jun 21, 2026 · Updated 24 times
Summary
This study tests whether cannabidiol (Epidiolex) can safely improve behavior, mood, sleep, and pain in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants aged 4 and older will receive either CBD or a placebo for 16 weeks, then switch after a break, followed by a year of open-label CBD. Caregivers will fill out surveys to track changes.
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This is a summary of
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
RECRUITINGTorrance, California, 90502, United States
Contact Email: •••••@•••••
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What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
cannabidiol (Epidiolex)
What this could lead to
If it works, this could point toward a way to manage difficult behaviors, mood, and sleep problems in Sanfilippo syndrome, improving daily life for patients and caregivers.
What could go wrong
This is a small, early-stage trial with only 35 participants, so results may not apply to everyone. Cannabidiol may cause side effects like drowsiness or liver issues, and the placebo-controlled design means some may not benefit during the blinded phase.
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.