Study of Cannabidiol in Sanfilippo Syndrome
Recruiting
Phase 2
Phase 3
Interventional Study
Sanfilippo Syndrome
Mucopolysaccharidosis III
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 and older
- Sex
- Any
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 35 (estimated)
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Other
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About This Trial
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the neurobehavioral symptoms and functional outcomes of Sanfilippo syndrome.
Each participant's caregiver will be asked to complete surveys related to the parti…
Trial Locations
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Eligibility Criteria
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…
Contacts