Batten Disease — Trials, FDA Actions & Pivotal Research

11 active Batten Disease trials currently recruiting in the US, sourced from ClinicalTrials.gov.

Synced daily from openFDA and ClinicalTrials.gov. Last sync: June 12, 2026.

Recruiting trials by city

Cities where Batten Disease trials in our archive had recruiting sites. Each link lists every currently recruiting paid clinical trial at that location.

Columbus, OH La Jolla, CA

Active Batten Disease trials

Currently recruiting US trials matching Batten Disease, sourced live from ClinicalTrials.gov. We’ll add per-event articles here as those trials post results or hit primary completion.

Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3

for Juvenile Neuronal Ceroid Lipofuscinosis

Sponsor: Polaryx Therapeutics, Inc.

Sex: All·Age: 6 - 18

Code: NCT04637282·Phase 3·Not Yet Recruiting

Gene Therapy Trial for CLN6 Batten Disease

for CLN6

1 location in La Jolla

Sponsor: The Charlotte and Gwenyth Gray Foundation

Sex: All·Age: 4 Months+

Code: NCT07582484·Phase 1, Phase 2·Not Yet Recruiting

Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies

for Neuronal Ceroid Lipofuscinosis

Sponsor: IRCCS Fondazione Stella Maris

Sex: All·Age: All ages

Code: NCT06844877·Recruiting

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NYSCF Scientific Discovery Biobank

for ALS

1 location in New York

Sponsor: New York Stem Cell Foundation Research Institute

Sex: All·Age: 30 Days+

Code: NCT06203106·Recruiting

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Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

for Neuronal Ceroid Lipofuscinosis

Sponsor: Universitätsklinikum Hamburg-Eppendorf

Sex: All·Age: All ages

Code: NCT04613089·Recruiting

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Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

for Batten Disease

1 location in Columbus

Sponsor: Jessica Scherr

Sex: All·Age: All ages

Code: NCT03862274·Enrolling By Invitation

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

for Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)

1 location in Bethesda

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Sex: All·Age: 1 Week - 100

Code: NCT03307304·Recruiting

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UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

for Adrenoleukodystrophy

1 location in Durham

Sponsor: Joanne Kurtzberg, MD

Sex: All·Age: 1 Week - 22

Code: NCT02254863·Phase 1·Recruiting

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Inherited Retinal Degenerative Disease Registry

for Eye Diseases Hereditary

1 location in Columbia

Sponsor: Foundation Fighting Blindness

Sex: All·Age: All ages·Accepts healthy volunteers

Code: NCT02435940·Recruiting

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Longitudinal Study of Neurodegenerative Disorders

for MLD

1 location in Pittsburgh

Sponsor: University of Pittsburgh

Sex: All·Age: All ages

Code: NCT03333200·Recruiting

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Clinical and Neuropsychological Investigations in Batten Disease

for Neuronal Ceroid Lipofuscinosis

1 location in Rochester

Sponsor: University of Rochester

Sex: All·Age: All ages

Code: NCT01873924·Recruiting

Apply to this trial