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All 5 Sanfilippo Syndrome trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Longitudinal Study of Neurodegenerative DisordersOnly in PennsylvaniaRecruitingUniversity of Pittsburgh1US site
- Natural History Study of Participants With Sanfilippo Syndrome Type IIICOnly in TexasRecruitingAges 12 Months+Phoenix Nest1US site
- Study of Cannabidiol in Sanfilippo SyndromeOnly in CaliforniaRecruitingPhase 2/3Ages 4+Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center1US site
- MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingInstitut National de la Santé Et de la Recherche Médicale, FranceNo US site yet
- Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHRecruitingPhase 2/3Ultragenyx PharmaceuticalNo US site yet
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Who runs the Sanfilippo Syndrome trials in the US, and what stage they are in
Of the 5 Sanfilippo Syndrome trials with a US site, 1 is in Phase 3, the last stage before a company can ask the FDA for approval. One more is an earlier Phase 1 or 2 test of safety and dosing. The remaining 3 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 3 of them, and universities, hospitals, foundations and government groups run 2. Phoenix Nest runs the most, with 2. The most recently listed one first appeared on ClinicalTrials.gov on September 14, 2026.
Sanfilippo Syndrome trials in the US, state by state
All 5 Sanfilippo Syndrome trials with a US site accept children under 18. Their open sites are in 4 states, most often Texas, California and Missouri. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
US hospitals running the most Sanfilippo Syndrome trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Sanfilippo Syndrome.
- The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterTorrance, California1 trial
- The University of Texas Southwestern Medical CenterDallas, Texas1 trial
- UPMC Children's Hospital of PittsburghPittsburgh, Pennsylvania1 trial
- UT Southwestern Children's Medical CenterDallas, Texas1 trial
- Washington University School of Medicine Division of Genetics and Genomic Medicine, Rare Diseases Department of PediatricsSt Louis, Missouri1 trial
Help getting to a Sanfilippo Syndrome trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Sanfilippo Syndrome trial near you
Are there Sanfilippo Syndrome clinical trials near me?
As of November 13, 2026, 5 Sanfilippo Syndrome clinical trials are recruiting now and 4 are not yet recruiting. In the US, 5 of them have at least 1 open site, across 4 states. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Sanfilippo Syndrome clinical trial sites?
Recruiting Sanfilippo Syndrome trials list open sites in California, Missouri, Pennsylvania and Texas.
Are there Sanfilippo Syndrome trials for children near me?
All 5 Sanfilippo Syndrome trials with a US site accept children under 18. Their open sites are in 4 states, most often Texas, California and Missouri. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Sanfilippo Syndrome clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Sanfilippo Syndrome trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Sanfilippo Syndrome trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Sanfilippo Syndrome: Fayuvi (rebisufligene etisparvovec).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.