Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 20, 2026, 5:39 PM EDT

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Showing 25–27 of 27 matching trials from the live ClinicalTrials.gov search.
Conditions
Neuronal Ceroid Lipofuscinosis, Batten's Disease
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190, Intracerebroventricular (ICV) access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 16 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145
Conditions
Neuronal Ceroid Lipofuscinosis CLN5
Interventions
NGN-101
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years to 9 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Rochester, New York