National Neuromuscular Research Institute | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis	Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, generalized myasthenia gravis, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndrome	Recruiting	Not stated	2026-09-24		NCT07294170
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy	Becker muscular dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Enrolling by invitation	Phase 2	2026-09-17		NCT06066580
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Phase 2	2026-09-02		NCT07435129
Descartes-08 in Autoantibody Myositis	antisynthetase syndrome, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease	Recruiting	Phase 2	2026-08-17		NCT07391605
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant	Alzheimer disease, Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cognitive disorder, dementia, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, proteostasis deficiencies, psychiatric disorder, tauopathy	Recruiting	Phase 3	2026-08-04		NCT06563895
A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis	Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndrome	Active, not recruiting	Phase 3	2026-07-08		NCT05070858
A Non-interventional, Post-authorisation Safety Study of Patients Treated With Efgartigimod Alfa	Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndrome	Recruiting	Not stated	2026-02-17		NCT06298565