11 results · Page 1 of 1

Swipe the table sideways for more columns.

TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma PatientsHigh-Risk NeuroblastomaRecruitingPhase 2 / Phase 32026-10-08NCT07549321
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
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)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 disorderNot yet recruitingPhase 32026-08-14NCT07681713
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45Congenital, 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 diseaseRecruitingPhase 1 / Phase 22026-08-13NCT07038824
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44Congenital, 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 diseaseRecruitingPhase 1 / Phase 22026-08-13NCT07037862
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)Abnormalities, 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 32026-08-06NCT06617429
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin MutationsDeafness, Autosomal Recessive 9, Ear Diseases, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, auditory neuropathy, hearing disorder, hearing loss disorder, nervous system disorder, otorhinolaryngologic diseaseRecruitingPhase 1 / Phase 22026-08-05NCT05788536
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)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 diseaseNot yet recruitingPhase 22026-07-02NCT07682129
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular DystrophyCongenital, 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 22026-06-25NCT06280209
Safety, Tolerability, Pharmacokinetics, and Efficacy of Filgotinib for the Treatment of Polyarticular-course Juvenile Idiopathic Arthritis in Children and AdolescentsSkin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, immune system disorder, juvenile idiopathic arthritis, musculoskeletal system disorder, rheumatic disorderRecruitingPhase 32026-06-12NCT07554495
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaActive, not recruitingPhase 32026-03-13NCT03424018