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Showing 25–40 of 16 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Batten Disease Clear all
Recruiting Phase 1 Interventional

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

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 7, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
Terminated No phase listed Observational

Natural History Study of Batten Disease

NCT04644549
View directory record Official record
Conditions
Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN3
Interventions
Not listed
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
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 3, 2016 · Synced Jul 21, 2026, 2:27 PM EDT
Completed No phase listed Observational

A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)

NCT03822650
View directory record Official record
Conditions
Ceroid Lipofuscinosis, Neuronal 5
Interventions
Not listed
Lead sponsor
Neurogene Inc.
Industry
Eligibility
Not listed
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Jul 21, 2026, 2:27 PM EDT
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 29, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
Terminated Not applicable Interventional Results available

Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision

NCT04974307
View directory record Official record
Conditions
CLN3-related Disorders, Non-CLN3 Related Low Vision
Interventions
OrCam MyEye 2
Device
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Years to 18 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Jul 21, 2026, 2:27 PM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Jul 21, 2026, 2:27 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Does Batten Grafting Improve Nasal Outcomes in Septoplasty and Turbinate Reduction?

NCT05287841
View directory record Official record
Conditions
Nasal Obstruction, Septal Defect, Allergic Rhinitis, Nasal Polyps, Nasal Valve Collapse
Interventions
Batten batten graft, Septoplasty, Inferior Turbinate Reduction
Procedure
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
96 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
3
States / cities
Poplar Bluff, Missouri • New York, New York • Staten Island, New York
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Jul 21, 2026, 2:27 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa
Drug
Lead sponsor
David L Rogers, MD
Other
Eligibility
24 Months to 72 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Jul 21, 2026, 2:27 PM EDT
Active, not recruiting Phase 1 Interventional

Study for the Treatment for CLN7 Disease

NCT04737460
View directory record Official record
Conditions
CLN7
Interventions
AAV9/CLN7
Genetic
Lead sponsor
Benjamin Greenberg
Other
Eligibility
1 Year to 18 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 30, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Children With CLN3 Batten Disease

NCT03770572
View directory record Official record
Conditions
CLN3, Batten Disease
Interventions
Low dose CLN-301, High dose CLN-301
Genetic
Lead sponsor
Neela Therapeutics
Industry
Eligibility
3 Years to 10 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jul 21, 2026, 2:27 PM EDT
Enrolling by invitation No phase listed Observational

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

NCT03862274
View directory record Official record
Conditions
Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
Interventions
CLN2 Treatment
Other
Lead sponsor
Jessica Scherr
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
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 1, 2025 · Synced Jul 21, 2026, 2:27 PM EDT
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 20, 2019 · Synced Jul 21, 2026, 2:27 PM EDT
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 14, 2015 · Synced Jul 21, 2026, 2:27 PM EDT
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 8, 2026 · Synced Jul 21, 2026, 2:27 PM EDT