Batten Disease

24 registered clinical trials studyying Batten Disease — 8 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	NYSCF Scientific Discovery Biobank NCT06203106	New York Stem Cell Foundation Research Institute	—
Completed	An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batt NCT05174039	Beyond Batten Disease Foundation	Phase 1 / Phase 2
Terminated	Natural History Study of Batten Disease NCT04644549	Emily de los Reyes	—
Recruiting	Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Dat NCT04613089	Universitätsklinikum Hamburg-Eppendorf	—
Active Not Recruiting	Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer NCT04273243	Emily de los Reyes	—
Enrolling By Invitation	Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease NCT03862274	Jessica Scherr	—
Active Not Recruiting	Gene Therapy for Children With CLN3 Batten Disease NCT03770572	Alcyone Therapeutics, Inc	Phase 1 / Phase 2
Recruiting	Investigations of Juvenile Neuronal Ceroid Lipofuscinosis NCT03307304	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	—
Active Not Recruiting	Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae NCT03285425	Emily de los Reyes	—
Completed	A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Diseas NCT02678689	BioMarin Pharmaceutical	Phase 2
Completed	An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease NCT02485899	BioMarin Pharmaceutical	Phase 1 / Phase 2
Recruiting	UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte- NCT02254863	Joanne Kurtzberg, MD	Phase 1
Recruiting	Inherited Retinal Degenerative Disease Registry NCT02435940	Foundation Fighting Blindness	—
Completed	A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy NCT01907087	BioMarin Pharmaceutical	Phase 1 / Phase 2
Completed	Human Placental-Derived Stem Cell Transplantation NCT01586455	New York Medical College	Phase 1
Recruiting	Longitudinal Study of Neurodegenerative Disorders NCT03333200	University of Pittsburgh	—
Terminated	Collection of Cerebrospinal Fluid in Healthy Children NCT01698229	Weill Medical College of Cornell University	—
Completed	Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis NCT01161576	Weill Medical College of Cornell University	Phase 1
Completed	AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis NCT01414985	Weill Medical College of Cornell University	Phase 1 / Phase 2
Completed	Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis NCT01035424	Weill Medical College of Cornell University	—
Recruiting	Clinical and Neuropsychological Investigations in Batten Disease NCT01873924	University of Rochester	—
Completed	Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis NCT00151216	Weill Medical College of Cornell University	Phase 1
Completed	Genotype - Phenotype Correlations of LINCL NCT00151268	Weill Medical College of Cornell University	—
Completed	Stem Cell Transplant for Inborn Errors of Metabolism NCT00176904	Masonic Cancer Center, University of Minnesota	Phase 2 / Phase 3