Clinical trials
Sanfilippo Syndrome clinical trials
Below are recruiting sanfilippo syndrome clinical trials, each written for real people, not researchers. We’re tracking 6 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06333041RecruitingPhase 2/Phase 3
CBD study for Sanfilippo syndrome
This study tests whether a cannabis-derived medicine called cannabidiol (CBD) can help children with Sanfilippo syndrome, a rare genetic disorder. It may be a good fit for children who have already tried other treatments or whose disease is more advanced.
Torrance, CaliforniaAges 4 years+ - NCT05825131Recruiting
Natural history study for Sanfilippo type C disease
This study follows people with Sanfilippo syndrome type C to better understand how the disease changes over time. It does not appear to test a new medicine; instead, it collects health and development information that can help future treatments.
Dallas, TexasAges 1 year+ - NCT06567769RecruitingPhase 1
A study of GC1130A for Sanfilippo syndrome type A
This trial tests a new medicine called GC1130A for children with Sanfilippo syndrome type A (MPS IIIA). The goal is to see if it is safe and if it might help with symptoms.
Oakland, CaliforniaAges 1–18 - NCT02716246RecruitingPhase 2/Phase 3
Gene therapy for MPS IIIA to slow brain decline
This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
Columbus, OhioAges Any age - NCT04360265Enrolling by invitationPhase 3
Follow-up gene therapy study for MPS IIIA participants
This follow-up study looks at how a past gene therapy treatment (UX111) is affecting you over time. It mainly involves continued visits and safety checks, and it helps researchers understand long-term benefits and risks.
Columbus, OhioAges Any age - NCT06614894RecruitingPhase 2/Phase 3
High dose ambroxol for adults with Sanfilippo syndrome
This trial tests a high dose of a medicine called ambroxol in adults with MPS III (Sanfilippo syndrome). It aims to see if the drug is safe and can help symptoms.
Fairfax, VirginiaAges 18–99
Hear when a new Sanfilippo Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for sanfilippo syndrome?
- Yes. Clin2 currently lists 6 recruiting sanfilippo syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a sanfilippo syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a sanfilippo syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.