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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)Charcot-Marie-Tooth disease type 4JRecruitingPhase 1 / Phase 22026-09-09NCT07447557
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4Jnervous system disorder, neuromuscular disease, peripheral nervous system disorderActive, not recruitingNot stated2026-07-08NCT06151600
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Malformations of Cortical Development, Group I, Muscle Hypertonia, Muscle Spasticity, Musculoskeletal Abnormalities, Neurologic Manifestations, Neuromuscular Manifestations, Paraplegia, Spastic, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, central nervous system malformation, cerebral cortical dysplasia, hereditary spastic paraplegia 50, microcephaly, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, palsy, paraplegiaRecruitingPhase 1 / Phase 22026-07-08NCT05518188
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary spastic paraplegia 50, nervous system disorder, palsy, paraplegiaRecruitingPhase 32026-04-20NCT06692712
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Lafora disease, brain disorder, central nervous system disorder, epilepsy, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, progressive myoclonus epilepsyRecruitingPhase 1 / Phase 22025-12-18NCT06609889