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ClinicalTrials.gov public records Last synced May 21, 2026, 6:37 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 40 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Batten Disease Clear all
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 30, 2020 · Synced May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational
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 May 21, 2026, 6:37 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 28, 2020 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 16, 2025 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 6:37 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 1, 2021 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
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 Mar 29, 2026 · Synced May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational
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 Feb 23, 2026 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Batten Disease
Interventions
Miglustat 100 milligrams (mg) Oral Capsule
Drug
Lead sponsor
Beyond Batten Disease Foundation
Other
Eligibility
17 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 8, 2025 · Synced May 21, 2026, 6:37 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
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 May 21, 2026, 6:37 PM EDT
Completed Phase 1 Interventional
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 24, 2022 · Synced May 21, 2026, 6:37 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
1 Year and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced May 21, 2026, 6:37 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers
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 May 21, 2026, 6:37 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 21, 2026, 6:37 PM EDT
Recruiting No phase listed Observational
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 11, 2025 · Synced May 21, 2026, 6:37 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional
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 13, 2026 · Synced May 21, 2026, 6:37 PM EDT
Terminated No phase listed Observational
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 May 21, 2026, 6:37 PM EDT
Withdrawn Phase 1 Interventional
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis
Interventions
HuCNS-SC
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 6 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 14, 2015 · Synced May 21, 2026, 6:37 PM EDT
Active, not recruiting No phase listed Observational
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 May 21, 2026, 6:37 PM EDT
Active, not recruiting No phase listed Observational
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 May 21, 2026, 6:37 PM EDT
Completed Phase 1Phase 2 Interventional Results available
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 7, 2019 · Synced May 21, 2026, 6:37 PM EDT