Condition
Mucopolysaccharidosis III clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Mucopolysaccharidosis III clinical trials that are recruiting, at sites in 4 countries (most in United States (2), Australia (1), Spain (1)). By phase: Phase 2/3 (2). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 4Countries
Newest Mucopolysaccharidosis III clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Cannabidiol (Epidiolex) for neurobehavioral symptoms in Sanfilippo syndrome | Phase 2/3 | Torrance, California | Recruiting | Mar 27, 2024 |
| Observational study on Mucopolysaccharidosis diseases in France | — | Angers and 22 other locations | Recruiting | Sep 14, 2023 |
| Gene therapy for treating MPS IIIA | Phase 2/3 | Columbus, Ohio and 4 other locations | Recruiting | Mar 23, 2016 |
All recruiting trials (3)
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Cannabidiol (Epidiolex) for neurobehavioral symptoms in Sanfilippo syndrome
This trial will test whether Epidiolex (a pharmaceutical cannabidiol) is safe and can help children and young people with Sanfilippo syndrome reduce neurobehavioral problems and improve daily functioning.
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Observational study on Mucopolysaccharidosis diseases in France
This study is trying to learn more about Mucopolysaccharidosis diseases by collecting information from patients in France to understand how these rare conditions affect their lives.
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Gene therapy for treating MPS IIIA
This study is testing a new gene therapy called UX111 to see if it can help people with MPS IIIA, a serious genetic disorder.
Related topics
Quick answers
How many Mucopolysaccharidosis III clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 4 countries. The most sites are in United States (2 trials), Australia (1 trial), Spain (1 trial), France (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 2/3: 2. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.