Independent directory Public ClinicalTrials.gov records United States
Clear filters
ClinicalTrials.gov public records Last synced Sep 11, 2026, 10:01 AM EDT

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

Showing 25–36 of 12 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
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
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
Conditions
Infantile Neronal Ceroid Lipofuscinosis
Interventions
Cystagon
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 3 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Not yet recruiting Phase 1Phase 2 Interventional

Gene Therapy Trial for CLN6 Batten Disease

NCT07582484
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
Terminated No phase listed Observational Accepts healthy volunteers

Collection of Cerebrospinal Fluid in Healthy Children

NCT01698229
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
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
Recruiting No phase listed Observational Accepts healthy volunteers

Caregiving Networks Across Disease Context and the Life Course

NCT05007990
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
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
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
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
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
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