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All 5 Niemann-Pick Disease Type C trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Most sites in Minnesota, California and TexasRecruitingPhase 3Ages 4+Azafaros B.V.5US sites
4 states - A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 GangliosidosisMost sites in Minnesota, California and TexasRecruitingPhase 3Ages 4+Azafaros B.V.5US sites
4 states - Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type COnly in MarylandRecruitingAges 3 Months+Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)1US site
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type COnly in MarylandRecruitingAges 1 Day-120Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)1US site
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type CRecruitingPhase 3Ages 4+IntraBioNo US site yet
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Who runs the Niemann-Pick Disease Type C trials in the US, and what stage they are in
Of the 4 Niemann-Pick Disease Type C trials with a US site, 2 are in Phase 3, the last stage before a company can ask the FDA for approval. The remaining 2 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 2 of them, and universities, hospitals, foundations and government groups run 2. The sponsors with the most are Azafaros B.V. (2) and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (2). The most recently listed one first appeared on ClinicalTrials.gov on July 24, 2025.
Niemann-Pick Disease Type C trials in the US, state by state
All 4 Niemann-Pick Disease Type C trials with a US site accept children under 18. Their open sites are in 5 states, most often California, Maryland and Minnesota. 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 Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C.
- Children's Medical Center DallasDallas, Texas2 trials
- Lysosomal Rare Disorders Research and Treatment CenterFairfax, Virginia2 trials
- National Institutes of Health Clinical CenterBethesda, Maryland2 trials
- UCSF Children's Hospital and Research Center at OaklandOakland, California2 trials
- University of Minnesota Medical SchoolMinneapolis, Minnesota2 trials
- Mayo Clinic Children's Center - PINRochester, Minnesota1 trial
- Mayo Clinic Childrens Center - PINRochester, Minnesota1 trial
Help getting to a Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C trial near you
Are there Niemann-Pick Disease Type C clinical trials near me?
As of November 13, 2026, 5 Niemann-Pick Disease Type C clinical trials are recruiting now and 1 is not yet recruiting. In the US, 4 of them have at least 1 open site, across 5 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 Niemann-Pick Disease Type C clinical trial sites?
Recruiting Niemann-Pick Disease Type C trials list open sites in California, Maryland, Minnesota, Texas and Virginia.
Are there Niemann-Pick Disease Type C trials for children near me?
All 4 Niemann-Pick Disease Type C trials with a US site accept children under 18. Their open sites are in 5 states, most often California, Maryland and Minnesota. 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 Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C: Aqneursa (levacetylleucine), Miplyffa (arimoclomol).
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.