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ClinicalTrials.gov public records Last synced Nov 2, 2026, 6:55 PM EST

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Showing 25–37 of 37 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: CLN3 Clear all
Terminated No phase listed Observational Accepts healthy volunteers

Collection of Cerebrospinal Fluid in Healthy Children

NCT01698229
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
Group 1
Other
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 8 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 4, 2016 · Synced Nov 2, 2026, 6:55 PM EST
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
View directory record Official record
Conditions
Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
2,800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Nov 2, 2026, 6:55 PM EST
Completed Phase 2 Interventional Results available

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

NCT01399047
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis
Interventions
Mycophenolate mofetil, Liquid Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
6 Years to 25 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated May 21, 2019 · Synced Nov 2, 2026, 6:55 PM EST
Not yet recruiting Phase 1Phase 2 Interventional

Gene Therapy Trial for CLN6 Batten Disease

NCT07582484
View directory record Official record
Conditions
CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
Interventions
scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
Drug
Lead sponsor
The Charlotte and Gwenyth Gray Foundation
Other
Eligibility
4 Months and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Nov 2, 2026, 6:55 PM EST
Active, not recruiting No phase listed Observational

Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae

NCT03285425
View directory record Official record
Conditions
Batten Disease, CLN6
Interventions
Natural history
Other
Lead sponsor
Emily de los Reyes
Other
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Nov 2, 2026, 6:55 PM EST
Recruiting No phase listed Observational

Clinical and Neuropsychological Investigations in Batten Disease

NCT01873924
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2035
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2025 · Synced Nov 2, 2026, 6:55 PM EST
Completed Phase 1Phase 2 Interventional Results available

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

NCT01907087
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
Interventions
BMN 190
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 15 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 8, 2019 · Synced Nov 2, 2026, 6:55 PM EST
Not yet recruiting Phase 3 Interventional

Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease

NCT07882290
View directory record Official record
Conditions
CLN3
Interventions
Miglustat oral liquid formulation (250 mg/mL)
Drug
Lead sponsor
Beyond Batten Disease Foundation
Other
Eligibility
4 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Aurora, Colorado • Baltimore, Maryland • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Nov 2, 2026, 6:55 PM EST
Completed Phase 1 Interventional

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

NCT00337636
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis
Interventions
Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system
Procedure · Drug
Lead sponsor
StemCells, Inc.
Industry
Eligibility
18 Months to 12 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Nov 2, 2026, 6:55 PM EST
Active, not recruiting No phase listed Observational

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243
View directory record Official record
Conditions
CLN6, Batten Disease
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Nov 2, 2026, 6:55 PM EST
Recruiting No phase listed Observational

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

NCT03307304
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Week to 100 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2050
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Nov 2, 2026, 6:55 PM EST
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Nov 2, 2026, 6:55 PM EST
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Nov 2, 2026, 6:55 PM EST