inherited neurodegenerative disorder
inherited neurodegenerative disorder
Explore linked clinical trials. Standard reference: MONDO:0024237729 results ยท Page 1 of 30
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Mitochondrial Dysfunction, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder | Not yet recruiting | Phase 1 | 2026-10-09 | NCT07869654 | |
| Brain Iron in Beta-Propeller Protein Associated Neurodegeneration (BPAN) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nerve Degeneration, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, iron metabolism disease, metabolic disease, movement disorder, nervous system disorder, neuroaxonal dystrophy, neurodegeneration with brain iron accumulation, neurodegenerative disease, pantothenate kinase-associated neurodegeneration | Not yet recruiting | Not applicable | 2026-10-09 | NCT07868120 | |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 3 | 2026-10-09 | NCT07778836 | |
| A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, autism, autism spectrum disorder, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorder | Not yet recruiting | Phase 2 / Phase 3 | 2026-10-09 | NCT07439510 | |
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Phase 2 | 2026-10-09 | NCT06447025 | |
| Impact of a Cardiac Rehabilitation Program on Patients With Cardiac Amyloidosis | Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, cardiovascular disorder, familial amyloid neuropathy, heart disorder, heart failure, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficiencies | Recruiting | Not applicable | 2026-10-08 | NCT07494942 | |
| A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, central nervous system malformation, hereditary disease, hereditary sensory and autonomic neuropathy, hypertensive disorder, inherited neurodegenerative disorder, interstitial lung disease, lung disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorder, vascular disorder | Recruiting | Phase 2 | 2026-10-08 | NCT07175038 | |
| Investigating Exercise in Myotonic Dystrophy Type 2 (DM2) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonic Dystrophy 2, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not applicable | 2026-10-08 | NCT06716931 | |
| ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Rett syndrome, X-linked disease, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06430385 | |
| Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program | Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, familial amyloid neuropathy, 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 | Recruiting | Not stated | 2026-10-08 | NCT05040373 | |
| Deep Brain Stimulation Therapy in Movement Disorders | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Parkinson disease, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, essential tremor, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, parkinsonian disorder, psychiatric disorder, synucleinopathy, tic disorder | Recruiting | Not applicable | 2026-10-08 | NCT02119611 | |
| AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT01362803 | |
| Natural History Study of Patients With Neurofibromatosis Type I | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, connective tissue neoplasm, cranial nerve neoplasm, cranial nerve neuropathy, epithelial neoplasm, eye disorder, fibrosarcoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, optic nerve disorder, optic nerve glioma, optic nerve neoplasm, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, primitive neuroectodermal tumor, sarcoma | Active, not recruiting | Not stated | 2026-10-08 | NCT00924196 | |
| Study of Skin Tumors in Tuberous Sclerosis | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, central nervous system malformation, cerebral cortical dysplasia, hamartoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, tuberous sclerosis | Recruiting | Not stated | 2026-10-08 | NCT00001975 | |
| Role of Genetic Factors in the Development of Lung Disease | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fibrosis, Hemic and Lymphatic Diseases, Hypersensitivity, Delayed, Hypersensitivity, Immediate, Infant, Newborn, Diseases, Malformations of Cortical Development, Group I, Neoplasms, Multiple Primary, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Respiratory Hypersensitivity, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, asthma, bronchial disorder, central nervous system malformation, cerebral cortical dysplasia, cystic fibrosis, hamartoma, hereditary disease, hereditary neoplastic syndrome, immune system disorder, inherited neurodegenerative disorder, interstitial lung disease, liver disorder, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, obstructive lung disease, pancreas disorder, pulmonary emphysema, pulmonary fibrosis, pulmonary sarcoidosis, respiratory system disorder, sarcoidosis, tuberous sclerosis | Recruiting | Not stated | 2026-10-08 | NCT00001532 | |
| Study of the Disease Process of Lymphangioleiomyomatosis | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Malformations of Cortical Development, Group I, Neoplasm, Lymphatic Tissue, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Multiple Primary, central nervous system malformation, cerebral cortical dysplasia, hamartoma, hereditary disease, hereditary neoplastic syndrome, immune system disorder, immunoproliferative disorder, inherited neurodegenerative disorder, lung disorder, lymphangioleiomyomatosis, lymphangiomyoma, lymphatic system disorder, lymphoproliferative syndrome, neoplasm, neoplasm with perivascular epithelioid cell differentiation, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, pleural disorder, pneumothorax, respiratory system disorder, tuberous sclerosis | Recruiting | Not stated | 2026-10-08 | NCT00001465 | |
| Pridopidine Phase 3 Study in 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 | Recruiting | Phase 3 | 2026-10-07 | NCT07609108 | |
| Oral Health and Wilson's Disease: SOMAWI | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Wilson disease, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, liver disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease | Recruiting | Not stated | 2026-10-06 | NCT05444127 | |
| The United Kingdom National Registry for Myotonic Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-10-06 | NCT04003363 | |
| MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma | Active, not recruiting | Phase 2 | 2026-10-06 | NCT03962543 | |
| Biomarkers in Friedreich's Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Friedreich ataxia, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Not stated | 2026-10-06 | NCT02497534 | |
| Everolimus bAsed caLcineurin inhibiTor frEe immunosuppRession oNe Year AfTer lIver transplantatiON (ALTERNATION) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, central nervous system malformation, hereditary disease, hereditary sensory and autonomic neuropathy, inherited neurodegenerative disorder, liver disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy | Recruiting | Phase 3 | 2026-10-05 | NCT07739914 | |
| A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophy | Recruiting | Phase 3 | 2026-10-05 | NCT07723248 | |
| Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, primitive neuroectodermal tumor | Not yet recruiting | Phase 2 | 2026-10-05 | NCT07521657 | |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Congenital, 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 disease | Recruiting | Phase 3 | 2026-10-05 | NCT07486934 |