CBD trial aims to ease sanfilippo syndrome symptoms

NCT ID NCT06333041

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on symptoms and stress levels.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
cannabidiol (CBD) oral solution
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.
What could go wrong
This is a small, early-stage trial with only 35 people. It may not show clear benefits, and CBD might not work for everyone or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 35 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2026

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * MPS III diagnosis confirmed by genetic testing * ≥ 4 years of age * Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 16 years of age, assent must also be provided when cognitively possible. * If taking any of the following, no dose changes for the last 8 weeks: 1. Anakinra 2. Fluoxetine 3. Probiotic supplement * One of the following criteria are met: 1. Previous participation in a gene/cell therapy or enzyme restorative clinical trial. 2. Previously ineligible to participate in a gene/cell therapy or enzyme restorative clinical trial due to advanced disease state. 3. Functional age, as measured by the Vineland, is ≤ 0.5 chronological age Exclusion Criteria: Equitable selection will be used when choosing participants. An individual who meets any of the following criteria will be excluded from participation in this study: * Mutation known to cause slowly progressive disease * Taken any form of cannabis, including cannabidiol, in the last 8 weeks * Currently enrolled in another ongoing clinical trial * Concomitant use of any of the following therapies: * Rifampin * Diazepam (except for intermittent use as needed for treatment of a prolonged seizure episode) * Clobazam * Stiripentol * Everolimus, sirolimus, tacrolimus * Digoxin * Valproate * Recreational or medical Tetrahydrocannabinol (THC) or synthetic cannabinoid medications (including Sativex) within the last three months * Felbamate (if taking for less than one year) * Non-pharmacological therapies (e.g. ketogenic diet) must be stable for up to four weeks prior to enrollment * Clinical evidence of liver disease or liver injury as indicated by the presence of abnormal tests (AST or ALT \> 2 x ULN; Bilirubin \> 2 x ULN) * Known hypersensitivity to any components of Epidiolex (cannabidiol) * Pregnant or lactating women * Any other social or medical condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study

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Conditions

The condition(s) this trial relates to.

Mucopolysaccharidosis III mucopolysaccharidosis type 3

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

    RECRUITING

    Torrance, California, 90502, United States

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