Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Batten Disease: early-phase trial registry
5 of 12 indexed Batten Disease trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,429 of 28,707 conditions by registered trial count.
12 US clinical trials · 5 currently recruiting · #1,429 of 28,707 conditions by registered trial count
Batten Disease: 12 US clinical trials tracked, 5 recruiting.
Batten Disease is the subject of 12 registered US clinical trials on ClinicalTrials.gov, 5 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 5 in Phase 1-2 (earlier-stage). The most active sponsor is Emily de los Reyes, running 2 of these trials.
Enrollment posture brief
Registry condition batten-disease
OPEN 5 | RECR 42% | LATE 0 | EARLY 5 | RANK #1429 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Adiposity (42% recruiting · 12 trials)
Batten Disease lists 5 open slots (42% of 12 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Batten Disease on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Batten Disease is recruiting below its size-band peers.
5 of 12 indexed trials (41.7%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Batten Disease research.
Emily de los Reyes accounts for 2 of 12 trials (16.7%). 11 distinct sponsors appear on at least one study for this condition label.
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Typical Batten Disease enrolment sits around 48 participants.
Median target enrolment is 48 among the 12 trials that report a genuine target (sentinel values excluded). 7 of those (58.3%) plan for fewer than 100 participants.
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Batten Disease splits between interventional and observational designs.
5 interventional and 7 observational studies make up this index (41.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
12 total, page 1 of 1
Inherited Retinal Degenerative Disease Registry
Foundation Fighting Blindness
NCT02435940
NYSCF Scientific Discovery Biobank
New York Stem Cell Foundation Research Institute
NCT06203106
Clinical and Neuropsychological Investigations in Batten Disease
University of Rochester
NCT01873924
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NCT03307304
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Joanne Kurtzberg, MD
NCT02254863
Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
Emily de los Reyes
NCT03285425
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
Emily de los Reyes
NCT04273243
Gene Therapy for Children With CLN3 Batten Disease
Alcyone Therapeutics
NCT03770572
Stem Cell Transplant for Inborn Errors of Metabolism
Masonic Cancer Center, University of Minnesota
NCT00176904
Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University
NCT01035424
Human Placental-Derived Stem Cell Transplantation
New York Medical College
NCT01586455
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
BioMarin Pharmaceutical
NCT02678689
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Batten Disease records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Batten Disease trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Batten Disease page
12 registered trials, 5 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 5 Batten Disease trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Emily de los Reyes sponsors the most Batten Disease trials on record, review their full trial history before evaluating a specific study. See Emily de los Reyes's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Batten Disease condition card totals 12 registered trials; most-linked intervention Intracerebroventricular access device; top US site state Ohio. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.