neuronal ceroid lipofuscinosis 2 | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Hereditary Central Nervous System Demyelinating Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sandhoff disease, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, neuronal ceroid lipofuscinosis 2, sphingolipidosis	Not yet recruiting	Phase 2	2026-07-31		NCT07740512
Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 2	Active, not recruiting	Phase 1 / Phase 2	2026-04-30		NCT05152914
A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 2	Recruiting	Phase 1 / Phase 2	2025-12-18		NCT05791864
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 2	Enrolling by invitation	Not stated	2025-10-24		NCT03862274