| An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease (NCT05174039) | Beyond Batten Disease Foundation | 2022-03-10 |
| Natural History Study of Batten Disease (NCT04644549) | Emily de los Reyes | 2021-04-27 |
| Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database (NCT04613089) | Universitätsklinikum Hamburg-Eppendorf | 2020-04-08 |
| Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer (NCT04273243) | Emily de los Reyes | 2020-01-24 |
| Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease (NCT03862274) | Jessica Scherr | 2018-12-01 |
| Gene Therapy for Children With CLN3 Batten Disease (NCT03770572) | Neela Therapeutics | 2018-11-13 |
| Investigations of Juvenile Neuronal Ceroid Lipofuscinosis (NCT03307304) | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2017-11-27 |
| Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae (NCT03285425) | Emily de los Reyes | 2017-01-01 |
| A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease (NCT02678689) | BioMarin Pharmaceutical | 2016-01-22 |
| An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease (NCT02485899) | BioMarin Pharmaceutical | 2015-02-01 |
| Inherited Retinal Degenerative Disease Registry (NCT02435940) | Foundation Fighting Blindness | 2014-06-01 |
| 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) | BioMarin Pharmaceutical | 2013-09-01 |
| Human Placental-Derived Stem Cell Transplantation (NCT01586455) | New York Medical College | 2013-04-01 |
| Longitudinal Study of Neurodegenerative Disorders (NCT03333200) | University of Pittsburgh | 2012-01-11 |
| Collection of Cerebrospinal Fluid in Healthy Children (NCT01698229) | Weill Medical College of Cornell University | 2011-12-01 |
| Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) (NCT01161576) | Weill Medical College of Cornell University | 2010-08-19 |
| AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis (NCT01414985) | Weill Medical College of Cornell University | 2010-04-15 |
| Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis (NCT01035424) | Weill Medical College of Cornell University | 2009-06-01 |
| Clinical and Neuropsychological Investigations in Batten Disease (NCT01873924) | University of Rochester | 2004-08-01 |
| Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (NCT00151216) | Weill Medical College of Cornell University | 2004-06-01 |
| Genotype - Phenotype Correlations of LINCL (NCT00151268) | Weill Medical College of Cornell University | 2004-03-01 |
| Stem Cell Transplant for Inborn Errors of Metabolism (NCT00176904) | Masonic Cancer Center, University of Minnesota | 1995-01-01 |