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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
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 atrophyRecruitingPhase 32026-10-05NCT07723248
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer DrugsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, 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 atrophyActive, not recruitingPhase 1 / Phase 22026-10-02NCT07336446
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, 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 atrophyActive, not recruitingPhase 32026-09-30NCT06764485
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 1 / Phase 22026-09-29NCT07190300
pTVG-HP DNA Vaccine With or Without pTVG-AR DNA Vaccine and Pembrolizumab in Patients With Castration-Resistant, Metastatic Prostate CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasm Metastasis, Neoplasms by Site, Neoplastic Processes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, metastatic malignant neoplasm, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 22026-08-26NCT04090528
UK SMA Patient RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Spinal Muscular Atrophies of Childhood, X-linked disease, autosomal recessive distal spinal muscular atrophy 1, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-08-20NCT04292574
Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-08-13NCT04944940
Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity46 XY differences of sex development, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Kennedy disease, Male Urogenital Diseases, Urogenital Abnormalities, Urogenital Diseases, X-linked disease, androgen insensitivity syndrome, central nervous system disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-08-13NCT04708431
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsAtrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscle tissue disorder, muscular atrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-07-09NCT05335876
ALS/MND Natural History Study Data RepositoryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Nutritional and Metabolic Diseases, TDP-43 proteinopathy, X-linked disease, amyotrophic lateral sclerosis, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, progressive bulbar palsy, proteostasis deficiencies, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-05-20NCT05966038
Bicalutamide and Abemaciclib in Inoperable or Metastatic Androgen Receptor-positive Triple-negative Breast CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Neoplasms by Site, Skin and Connective Tissue Diseases, X-linked disease, breast disorder, breast neoplasm, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, skin disorder, spinal cord disorder, spinal muscular atrophy, triple-negative breast carcinomaRecruitingPhase 22026-04-01NCT06365788
International SBMA Project (KDA)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingNot stated2026-03-02NCT07443449
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford3-methylglutaconic aciduria type 5, 46 XY differences of sex development, 4p16.3 microduplication syndrome, Abnormalities, Multiple, Alagille syndrome, Alstrom syndrome, Amino Acid Transport Disorders, Inborn, Amnesia, Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation, Aortic Stenosis, Supravalvular, Arterial Occlusive Diseases, Ataxia-Telangiectasia Variant, Axenfeld-Rieger syndrome, Bacterial Infections and Mycoses, Beckwith-Wiedemann syndrome, Blau syndrome, Bohring-Opitz syndrome, Bone Diseases, Developmental, Bone Diseases, Metabolic, Borrelia infectious disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cayman type cerebellar ataxia, Charlevoix-Saguenay spastic ataxia, Chronic Disease, Ciliopathies, Cockayne syndrome, Coffin-Lowry syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, DNA repair disease, De Lange Syndrome, Deafness, Deglutition Disorders, Denys-Drash syndrome, Digestive System Abnormalities, Digestive System Diseases, Digestive System Fistula, Disease Attributes, Disorders of Excessive Somnolence, Dwarfism, Dyskinesias, Dyssomnias, Ear Diseases, Emanuel syndrome, Eosinophilic enteropathy, Eye Abnormalities, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fibrosis, Fistula, Frasier syndrome, Friedreich ataxia, Frontotemporal Lobar Degeneration, Growth Deficiency and Mental Retardation with Facial Dysmorphism, Halitosis, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Hirschsprung disease, Hyperglycinemia, Nonketotic, Hypophosphatasia, Adult, Hypophosphatasia, Perinatal Lethal, Imprinting Disorders, Infections, KBG syndrome, Kabuki syndrome, Kawasaki disease, Kennedy disease, Kleine-Levin syndrome, Klippel-Feil syndrome, Kyphosis, Lambert-Eaton myasthenic syndrome, Leber congenital amaurosis, Leigh syndrome, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lyme disease, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Machado-Joseph disease, Machado-Joseph disease type 1, Machado-Joseph disease type 2, Machado-Joseph disease type 3, Malan overgrowth syndrome, Male Urogenital Diseases, Mannosidase Deficiency Diseases, Memory Disorders, Moyamoya disease, Muscle Hypertonia, Muscle Spasticity, Muscular Atrophy, Ataxia, Retinitis Pigmentosa, and Diabetes Mellitus, Muscular Disorders, Atrophic, Musculoskeletal Abnormalities, Myhre syndrome, Myoclonus, Myoclonus, Nocturnal, NARP syndrome, Neglected Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Multiple Primary, Neurobehavioral Manifestations, Neuroinflammatory Diseases, Neurologic Manifestations, Neuromuscular Manifestations, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, Olivary Degeneration, Optic Atrophy, Autosomal Dominant, PHARC syndrome, Paraneoplastic Syndromes, Nervous System, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Peters anomaly, Pitt-Hopkins syndrome, Postoperative Complications, Potocki-Shaffer syndrome, Primary Immunodeficiency Diseases, Pyruvate Dehydrogenase Complex Deficiency Disease, Pyruvate Metabolism, Inborn Errors, Rare Diseases, Sacral agenesis, Sacral defect and anterior sacral meningocele, Scheuermann disease, Sensation Disorders, Signs and Symptoms, Signs and Symptoms, Digestive, Skin and Connective Tissue Diseases, Skraban-Deardorff syndrome, Sleep Disorders, Intrinsic, Spinal Curvatures, Spinal Osteochondrosis, Spinocerebellar Ataxia, Autosomal Recessive 8, Spinocerebellar Ataxias, Spinocerebellar Degenerations, Spinocerebellar ataxia 8, Stickler syndrome type 1, Stickler syndrome type 2, Sulfatidosis, TDP-43 proteinopathy, Urogenital Abnormalities, Urogenital Diseases, Urogenital Neoplasms, WAGR syndrome, Warburg Sjo Fledelius syndrome, Wiedemann-Steiner syndrome, Williams syndrome, Wilms tumor, Wolf-Hirschhorn syndrome, X-linked cerebellar ataxia, X-linked disease, X-linked intellectual disability-cerebellar hypoplasia syndrome, X-linked non progressive cerebellar ataxia, X-linked progressive cerebellar ataxia, X-linked spinocerebellar ataxia type 3, X-linked spinocerebellar ataxia type 4, achalasia, acquired ataxia, acrodysostosis, adrenal gland disorder, adrenocortical insufficiency, adult Refsum disease, adult hypophosphatasia, anal fistula, anemia, aniridia, aniridia-absent patella syndrome, aortic valve disorder, aortic valve stenosis, arginase deficiency, atactic disorder, non-human animal, ataxia neuropathy spectrum, ataxia telangiectasia, ataxia, early-onset, with oculomotor apraxia and hypoalbuminemia, ataxia-telangiectasia-like disorder, atypical hemolytic-uremic syndrome, autoimmune disease, autoimmune disorder of the nervous system, autonomic nervous system disorder, autosomal dominant cerebellar ataxia, autosomal dominant cerebellar ataxia type I, autosomal dominant cerebellar ataxia type III, autosomal dominant cerebellar ataxia type IV, autosomal dominant cerebellar ataxia, deafness and narcolepsy, autosomal dominant spastic ataxia, autosomal recessive ataxia due to PEX10 deficiency, autosomal recessive ataxia due to ubiquinone deficiency, autosomal recessive ataxia, Beauce type, autosomal recessive cerebellar ataxia, autosomal recessive cerebellar ataxia with late-onset spasticity, autosomal recessive congenital cerebellar ataxia, autosomal recessive degenerative and progressive cerebellar ataxia, autosomal recessive metabolic cerebellar ataxia, autosomal recessive spastic ataxia, autosomal recessive spinocerebellar ataxia 12, autosomal recessive spinocerebellar ataxia 7, autosomal recessive syndromic cerebellar ataxia, bacterial infectious disease, basal ganglia disorder, benign paroxysmal tonic upgaze of childhood with ataxia, benign urinary system neoplasm, beta-mannosidosis, bile duct disorder, biliary atresia, biliary tract disorder, blood platelet disease, bone disorder, brachydactyly-nystagmus-cerebellar ataxia syndrome, brain disorder, breast implant-associated anaplastic large cell lymphoma, cardiovascular disorder, carotid artery disorder, cataract-ataxia-deafness syndrome, cauda equina syndrome, caudal regression sequence, central nervous system disorder, central nervous system infectious disorder, central nervous system malformation, cerebellar ataxia, cerebellar ataxia-areflexia-pes cavus-optic atrophy-sensorineural hearing loss syndrome, cerebellar ataxia-ectodermal dysplasia syndrome, cerebellar ataxia-hypogonadism syndrome, cerebellar disorder, cerebral arterial disease, cerebrovascular disorder, childhood hypophosphatasia, cholestasis, chromosomal disorder, chromosome 1p36 deletion syndrome, chronic recurrent multifocal osteomyelitis, chronic renal failure syndrome, cirrhosis of liver, cognitive disorder, colonic disorder, congenital heart disease, congenital sucrase-isomaltase deficiency, connective tissue disorder, cranial nerve neuropathy, cystinosis, dementia, demyelinating disease, diffuse scleroderma, digestive system disorder, dimethylglycine dehydrogenase deficiency, disease, disorder of glycogen metabolism, disorder of sexual differentiation, dyskinesia of esophagus, dysostosis, early-onset cerebellar ataxia with retained tendon reflexes, endocrine system disorder, eosinophilic gastroenteritis, episodic ataxia type 1, episodic ataxia type 2, episodic ataxia type 3, episodic ataxia type 4, episodic ataxia type 5, episodic ataxia type 6, episodic ataxia type 7, esophageal disorder, eye disorder, familial caudal dysgenesis, familial isolated deficiency of vitamin E, fragile X-associated tremor/ataxia syndrome, frontotemporal dementia, gonadal disorder, gram-negative bacterial infections, hearing disorder, hearing loss disorder, heart disorder, heart valve disorder, hematologic disorder, hemolytic anemia, hemolytic-uremic syndrome, hereditary ataxia, hereditary disease, hereditary episodic ataxia, hereditary motor and sensory neuropathy, hereditary neoplastic syndrome, hereditary optic atrophy, hereditary sensory and autonomic neuropathy, hereditary sensory neuropathy-deafness-dementia syndrome, histiocytosis, hyperacusis, hypertrophic olivary degeneration, hypophosphatasia, idiopathic achalasia, idiopathic gastroparesis, idiopathic hypersomnia, idiopathic hypersomnia with long sleep time, idiopathic hypersomnia without long sleep time, ileus, immune system disorder, inborn carbohydrate metabolic disorder, inborn disorder of amino acid metabolism, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inborn mitochondrial metabolism disorder, inclusion body myopathy with Paget disease of bone and frontotemporal dementia type 1, infantile hypophosphatasia, inherited neurodegenerative disorder, inherited retinal dystrophy, intellectual disability, intellectual disability-sparse hair-brachydactyly syndrome, intestinal disorder, intestinal fistula, intestinal obstruction, intestinal pseudo-obstruction, intracranial arterial disease, intrahepatic cholestasis, iris disorder, isolated aniridia, juvenile myasthenia gravis, juvenile nephropathic cystinosis, kidney cancer, kidney disorder, kidney failure, laryngeal papillomatosis, leiomyosarcoma, leiomyosarcoma of the cervix uteri, leiomyosarcoma of the corpus uteri, leukodystrophy, leukoencephalopathy with calcifications and cysts, liver disorder, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, malabsorption syndrome, malignant endocrine neoplasm, maternally-inherited Leigh syndrome, megacolon, meningitis, metabolic disease, metachromatic leukodystrophy, metaphyseal chondrodysplasia, Jansen type, mismatch repair cancer syndrome 1, mixed neoplasm, motor neuron disorder, movement disorder, mucolipidosis, mucolipidosis type IV, multiple endocrine neoplasia, multiple endocrine neoplasia type 1, multiple endocrine neoplasia type 2A, multiple endocrine neoplasia type 2B, multiple endocrine neoplasia type 4, multiple system atrophy, multiple system atrophy, cerebellar type, multiple system atrophy, parkinsonian type, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myasthenia gravis, myoclonus-cerebellar ataxia-deafness syndrome, myomatous neoplasm, myopathy, myofibrillar, 9, with early respiratory failure, narcolepsy, narcolepsy without cataplexy, neonatal myasthenia gravis, neoplasm, nephropathic cystinosis, nephropathic infantile cystinosis, nerve compression syndrome, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, non-Langerhans cell histiocytosis, oculocerebrorenal syndrome, oculopharyngeal muscular dystrophy, odontohypophosphatasia, optic atrophy, optic nerve disorder, osteochondrosis, otorhinolaryngologic disease, paraneoplastic syndrome, perinatal lethal hypophosphatasia, peripheral nervous system disorder, peroxisome biogenesis disorder 4B, polyneuropathy, polyradiculoneuropathy, prenatal benign hypophosphatasia, primary biliary cholangitis, primary dysautonomia, proteostasis deficiencies, psychiatric disorder, recessive mitochondrial ataxia syndrome, rectal disorder, recurrent respiratory papillomatosis, renal tubular transport disease, retinal degeneration, retinal disorder, retinitis pigmentosa, sarcoma, scleroderma, short bowel syndrome, silver-Russell syndrome due to 11p15 microduplication, silver-Russell syndrome due to maternal uniparental disomy of chromosome 11, skin disorder, skin vascular disease, sleep-wake disorder, small intestine leiomyosarcoma, spastic ataxia, spastic ataxia 3, spastic ataxia 7, spasticity-ataxia-gait anomalies syndrome, sphingolipidosis, spinal cord disorder, spinal muscular atrophy, spinocerebellar ataxia 7, spinocerebellar ataxia type 1, spinocerebellar ataxia type 10, spinocerebellar ataxia type 11, spinocerebellar ataxia type 12, spinocerebellar ataxia type 13, spinocerebellar ataxia type 14, spinocerebellar ataxia type 15/16, spinocerebellar ataxia type 17, spinocerebellar ataxia type 18, spinocerebellar ataxia type 19/22, spinocerebellar ataxia type 2, spinocerebellar ataxia type 20, spinocerebellar ataxia type 21, spinocerebellar ataxia type 23, spinocerebellar ataxia type 25, spinocerebellar ataxia type 26, spinocerebellar ataxia type 27, spinocerebellar ataxia type 28, spinocerebellar ataxia type 29, spinocerebellar ataxia type 30, spinocerebellar ataxia type 31, spinocerebellar ataxia type 32, spinocerebellar ataxia type 34, spinocerebellar ataxia type 35, spinocerebellar ataxia type 36, spinocerebellar ataxia type 37, spinocerebellar ataxia type 4, spinocerebellar ataxia type 5, spinocerebellar ataxia type 6, spinocerebellar ataxia type 8, spinocerebellar ataxia with epilepsy, spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 1, spinocerebellar ataxia, autosomal recessive, with axonal neuropathy 2, spirochaetales infections, sporadic adult-onset ataxia of unknown etiology, syndromic disease, synucleinopathy, systemic sclerosis, telangiectasis, thrombocytopenia, thrombotic microangiopathy, tick-borne infectious disease, transient global amnesia, triple-A syndrome, urea cycle disorder, uremia, urinary system disorder, uveal disorder, vascular disorder, vasculitis, vector-borne disease, vertebral column disorder, viral infection of central nervous system, viral infectious disease, viral meningitisRecruitingNot stated2025-05-29NCT01793168
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 2 / Phase 32025-04-16NCT06862596
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 22024-04-15NCT06169046