Batten Disease — Trials, FDA Actions & Pivotal Research

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

Synced daily from openFDA and ClinicalTrials.gov. Last sync: August 27, 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.

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·Listed May 2026

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·Listed Feb 2025

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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·Listed Jan 2024

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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·Listed Nov 2020

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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·Listed Mar 2019

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·Listed Oct 2017

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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·Listed Oct 2014

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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·Listed May 2015

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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·Listed Nov 2017

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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·Listed Jun 2013

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