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.
Ready to participate?

Review the details below, then apply to join this clinical 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
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
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
Loading…

Loading trial locations…

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

Jonathan Acevedo, BS

310-357-9023

jonathan.acevedo@lundquist.org

CONTACT

Adolfo Morales, BA

310-357-9023

adolfo.morales@lundquist.org

CONTACT