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All 10 Batten Disease trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Caregiving Networks Across Disease Context and the Life CourseOnly in MarylandRecruitingAges 18-100National Human Genome Research Institute (NHGRI)1US site
- Clinical and Neuropsychological Investigations in Batten DiseaseOnly in New YorkRecruitingUniversity of Rochester1US site
- Inherited Retinal Degenerative Disease RegistryOnly in MarylandRecruitingFoundation Fighting Blindness1US site
- Investigations of Juvenile Neuronal Ceroid LipofuscinosisOnly in MarylandRecruitingAges 1 Week-100Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)1US site
- Longitudinal Study of Neurodegenerative DisordersOnly in PennsylvaniaRecruitingUniversity of Pittsburgh1US site
- NYSCF Scientific Discovery BiobankOnly in New YorkRecruitingAges 30 Days+New York Stem Cell Foundation Research Institute1US site
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsOnly in North CarolinaRecruitingPhase 1Ages 1 Week-22Joanne Kurtzberg, MD1US site
- A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 DiseaseRecruitingPhase 1/2Ages 24 Months-84 MonthsTern TherapeuticsNo US site yet
- Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic StrategiesRecruitingIRCCS Fondazione Stella MarisNo US site yet
- Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD DatabaseRecruitingUniversitätsklinikum Hamburg-EppendorfNo US site yet
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- Trial Alerts, when a new trial opens or a trial changes
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Who runs the Batten Disease trials in the US, and what stage they are in
None of the 8 Batten Disease trials with a US site is in Phase 3, the last stage before a company can ask the FDA for approval. Earlier Phase 1 or 2 tests of safety and dosing make up 2 of them. The remaining 6 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Universities, hospitals, foundations and government groups run all of them, with no company leading one. The most recently listed one first appeared on ClinicalTrials.gov on May 13, 2026.
Batten Disease trials in the US, state by state
Of the 8 Batten Disease trials with a US site, 7 accept children under 18. Their open sites are in 5 states, most often Maryland, New York and California. 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 Batten Disease 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 Batten Disease.
- National Institutes of Health Clinical CenterBethesda, Maryland2 trials
- Duke University Medical CenterDurham, North Carolina1 trial
- Foundation Fighting BlindnessColumbia, Maryland1 trial
- New York Stem Cell Foundation Research InstituteNew York, New York1 trial
- University of California, San Diego - Rady Children'sLa Jolla, California1 trial
- University of RochesterRochester, New York1 trial
- UPMC Children's Hospital of PittsburghPittsburgh, Pennsylvania1 trial
Help getting to a Batten Disease 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 Batten Disease trial near you
Are there Batten Disease clinical trials near me?
As of November 13, 2026, 10 Batten Disease clinical trials are recruiting now and 1 is not yet recruiting. In the US, 8 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 Batten Disease clinical trial sites?
Recruiting Batten Disease trials list open sites in California, Maryland, New York, North Carolina and Pennsylvania.
Are there Batten Disease trials for children near me?
Of the 8 Batten Disease trials with a US site, 7 accept children under 18. Their open sites are in 5 states, most often Maryland, New York and California. 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 Batten Disease 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 Batten Disease 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 Batten Disease 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 Batten Disease: Brineura (cerliponase alfa).
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.