Batten Disease Clinical Trials

25 clinical trials for Batten Disease across international registries, including 8 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Inherited Retinal Degenerative Disease Registry — RECRUITING
• Investigations of Juvenile Neuronal Ceroid Lipofuscinosis — RECRUITING
• Longitudinal Study of Neurodegenerative Disorders — RECRUITING
• Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease — RECRUITING
• Clinical and Neuropsychological Investigations in Batten Disease — RECRUITING
• UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells — RECRUITING · PHASE1
• NYSCF Scientific Discovery Biobank — RECRUITING
• Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database — RECRUITING
• Gene Therapy for Children With CLN3 Batten Disease — ACTIVE_NOT_RECRUITING · PHASE1
• Gene Therapy Trial for CLN6 Batten Disease — NOT_YET_RECRUITING · PHASE1
• Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer — ACTIVE_NOT_RECRUITING
• An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease — COMPLETED · PHASE1
• Natural History Study of Batten Disease — TERMINATED
• Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae — ACTIVE_NOT_RECRUITING
• A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease — COMPLETED · PHASE2
• Human Placental-Derived Stem Cell Transplantation — COMPLETED · PHASE1
• An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease — COMPLETED · PHASE1
• Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) — COMPLETED · PHASE1
• AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis — COMPLETED · PHASE1
• Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis — COMPLETED
• Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis — COMPLETED · PHASE1
• 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 — COMPLETED · PHASE1
• Stem Cell Transplant for Inborn Errors of Metabolism — COMPLETED · PHASE2
• Genotype - Phenotype Correlations of LINCL — COMPLETED
• Collection of Cerebrospinal Fluid in Healthy Children — TERMINATED

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