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Showing 1–16 of 16 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Jansky-Bielschowsky Disease Clear all
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 11, 2025 · Synced Jul 21, 2026, 10:10 PM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899
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, 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
Source: ClinicalTrials.gov public record
Updated Aug 23, 2022 · Synced Jul 21, 2026, 10:10 PM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

NCT01238315
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 Jul 21, 2026, 10:10 PM EDT
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
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 Jun 29, 2026 · Synced Jul 21, 2026, 10:10 PM EDT
Completed Phase 1Phase 2 Interventional

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01414985
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 Jul 21, 2026, 10:10 PM EDT
Completed Phase 2 Interventional Results available

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689
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 Jul 21, 2026, 10:10 PM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCT01161576
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 Jul 21, 2026, 10:10 PM EDT
Completed No phase listed Observational

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01035424
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 Jul 21, 2026, 10:10 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580
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 Jul 21, 2026, 10:10 PM EDT
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 7, 2019 · Synced Jul 21, 2026, 10:10 PM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT00151216
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAV2CUhCLN2 (3x10^12 particle units)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 23, 2020 · Synced Jul 21, 2026, 10:10 PM EDT
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 13, 2026 · Synced Jul 21, 2026, 10:10 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, 10:10 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, 10:10 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, 10:10 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, 10:10 PM EDT