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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseRecruitingPhase 32026-10-05NCT07486934
Long-term Safety and Efficacy Study of Riliprubart in Participants With CIDPChronic Disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, autoimmune disease, autoimmune disorder of the nervous system, chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating disease, immune system disorder, nervous system disorder, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, polyradiculoneuropathyEnrolling by invitationPhase 32026-09-30NCT06859099
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
A Study to Investigate Efficacy and Safety of SAR441566 in Patients With Ulcerative ColitisDigestive System Diseases, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, ulcerative colitisActive, not recruitingPhase 22026-09-25NCT06867094
A Study to Evaluate the Efficacy and Safety of KarXT for the Treatment of Manic Episodes in Bipolar-I DisorderMania, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nervous system disorderRecruitingPhase 32026-09-22NCT06951698
A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic Disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, autoimmune disease, autoimmune disorder of the nervous system, chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating disease, immune system disorder, nervous system disorder, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, polyradiculoneuropathyRecruitingPhase 32026-09-22NCT06290141
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)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 diseaseActive, not recruitingPhase 22026-09-15NCT05996003
A Study to Assess the Long-term Safety of KarXT for the Treatment of Manic Episodes in Bipolar-I Disorder (BALSAM-3)Mania, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nervous system disorderRecruitingPhase 32026-09-14NCT06929273
A Study of Vortioxetine in Japanese Pediatric Patients With Major Depressive Disorderdepressive disorder, major depressive disorder, mood disorder, psychiatric disorderRecruitingPhase 32026-09-02NCT07204314
Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis (RMS) (REMODEL-2)Charcot-Marie-Tooth disease type 1F, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, RecurrenceActive, not recruitingPhase 32026-08-18NCT05156281
A Study to Test the Effects and Safety of Riliprubart in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) for Which the Usual Treatments do Not WorkChronic Disease, Disease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, autoimmune disease, autoimmune disorder of the nervous system, chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating disease, immune system disorder, nervous system disorder, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, polyradiculoneuropathyActive, not recruitingPhase 32026-08-10NCT06290128
Pemafibrate for Symptomatic ICAS RCTDyslipidemias, Lipid Metabolism Disorders, Nutritional and Metabolic Diseases, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hyperlipidemia, hypertriglyceridemia, metabolic disease, nervous system disorder, stroke disorder, vascular disorderRecruitingNot applicable2026-08-03NCT07562178
Lung Insufflation Capacity Training and Respiratory Function in Amyotrophic Lateral SclerosisNutritional 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 disorderRecruitingNot applicable2026-07-30NCT07257302
A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear PalsyFrontotemporal Lobar Degeneration, Neurologic Manifestations, Ocular Motility Disorders, Pathological Conditions, Signs and Symptoms, Pick disease, Signs and Symptoms, basal ganglia disorder, brain disorder, central nervous system disorder, cognitive disorder, cranial nerve neuropathy, dementia, eye disorder, frontotemporal dementia, movement disorder, nervous system disorder, neurodegenerative disease, ophthalmoplegia, palsy, psychiatric disorder, tauopathyRecruitingPhase 32026-07-29NCT07498426
A Study to Evaluate the Efficacy and Safety of KarXT in Acutely Psychotic Japanese Adult Participants With SchizophreniaSchizophrenia Spectrum and Other Psychotic Disorders, psychiatric disorder, schizophreniaRecruitingPhase 32026-07-27NCT06882785
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)Behcet disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Purpura, Thrombocytopenic, Idiopathic, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anterior uveitis, antiphospholipid syndrome, autoimmune disease, blood coagulation disease, blood platelet disease, bullous pemphigoid, cardiovascular disorder, connective tissue disorder, dermatomyositis, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary periodic fever syndrome, hereditary skin disorder, immune system disorder, mouth disorder, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, panuveitis, polymyositis, purpura, skin disorder, skin vascular disease, thrombocytopenia, thrombocytopenic purpura, thrombotic microangiopathy, uveal disorder, uveitis, vascular disorder, vasculitis, vesiculobullous skin diseaseRecruitingPhase 12026-07-22NCT06371417
Efficacy and Safety Studies of Frexalimab (SAR441344) in Adults With Relapsing Forms of Multiple SclerosisCNS demyelinating autoimmune disease, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderActive, not recruitingPhase 32026-07-20NCT06141473
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)Behcet disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Purpura, Thrombocytopenic, Idiopathic, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anterior uveitis, antiphospholipid syndrome, autoimmune disease, blood coagulation disease, blood platelet disease, bullous pemphigoid, cardiovascular disorder, connective tissue disorder, dermatomyositis, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary periodic fever syndrome, hereditary skin disorder, immune system disorder, mouth disorder, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, panuveitis, polymyositis, purpura, skin disorder, skin vascular disease, thrombocytopenia, thrombocytopenic purpura, thrombotic microangiopathy, uveal disorder, uveitis, vascular disorder, vasculitis, vesiculobullous skin diseaseEnrolling by invitationPhase 12026-07-17NCT06723106
A Clinical Trial to Learn About the Effects of VHB937 in People With Early Alzheimer's DiseaseAlzheimer disease, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyRecruitingPhase 22026-06-26NCT07094516
A Study to Evaluate the Efficacy and Safety of Adjunctive KarXT for the Treatment of Mania, With or Without Mixed Features, in Participants With Bipolar-I Disorder Taking Lithium, Valproate, or LamotrigineBipolar and Related Disorders, Mania, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, bipolar disorder, mood disorder, nervous system disorder, psychiatric disorderRecruitingPhase 32026-06-23NCT07140913
NS-050/NCNP-03 in Boys With DMD (Meteor50)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 diseaseActive, not recruitingPhase 1 / Phase 22026-06-16NCT06053814
Long-term Safety, Tolerability and Effectiveness Study of Ofatumumab in Patients With Relapsing MSRelapsing Multiple SclerosisActive, not recruitingPhase 32026-05-27NCT03650114
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)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 diseaseActive, not recruitingPhase 32026-05-22NCT05881408
Efficacy and Safety Study of Frexalimab (SAR441344) in Adults With Nonrelapsing Secondary Progressive Multiple SclerosisCNS demyelinating autoimmune disease, autoimmune disease, autoimmune disorder of the nervous system, demyelinating disease, immune system disorder, multiple sclerosis, nervous system disorderActive, not recruitingPhase 32026-05-14NCT06141486
MSA-01 in Multiple System Atrophyautonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, primary dysautonomia, synucleinopathyRecruitingPhase 32026-03-04NCT07446894