UniQure Biopharma B.V. | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorder	Recruiting	Phase 1 / Phase 2	2026-09-24		NCT06270316
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy	Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Hippocampal Sclerosis, Malformations of Cortical Development, Group I, brain disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, epilepsy, nervous system disorder	Recruiting	Phase 1 / Phase 2	2026-08-24		NCT06063850
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)	Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder	Active, not recruiting	Phase 1 / Phase 2	2025-10-22		NCT06100276
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease	Cognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Huntington disease, basal ganglia disorder, brain disorder, central nervous system disorder, choreatic disease, cognitive disorder, dementia, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, psychiatric disorder	Active, not recruiting	Phase 1 / Phase 2	2025-10-21		NCT04120493
Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease	Cognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Huntington disease, basal ganglia disorder, brain disorder, central nervous system disorder, choreatic disease, cognitive disorder, dementia, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, psychiatric disorder	Active, not recruiting	Phase 1 / Phase 2	2025-03-10		NCT05243017