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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pharmacokinetics, Safety, and Efficacy of Povorcitinib in Adolescent Participants With Moderate to Severe Hidradenitis SuppurativaBacterial Infections and Mycoses, Infections, Skin and Connective Tissue Diseases, Suppuration, bacterial infectious disease, hidradenitis, hidradenitis suppurativa, skin disease caused by bacterial infection, skin disorder, skin disorder caused by infection, sweat gland disorderRecruitingPhase 22026-10-09NCT07213973
A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderRecruitingPhase 32026-09-25NCT05923073
A Study of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN)Hemolytic Disease of the Fetus and NewbornRecruitingPhase 32026-09-25NCT05912517
A Study of Ocrelizumab in Children and Adolescents With Relapsing-Remitting 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 22026-09-14NCT04075266
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 2 / Phase 32026-09-09NCT05115110
A U.S. Registry of Eosinophilic Esophagitis Pediatric, Adolescent and Adult Patients Treated With DUPIXENT® As Standard of CareDigestive System Diseases, Eosinophilia, Hemic and Lymphatic Diseases, Hypersensitivity, Immediate, digestive system disorder, eosinophilic esophagitis, esophageal disorder, esophagitis, gastroenteritis, hematologic disorder, immune system disorder, leukocyte disorderActive, not recruitingNot stated2026-08-12NCT06693531
Nucleoside Therapy in Patients With Telomere Biology DisordersBone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fibrosis, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Revesz syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, bone marrow disorder, dyskeratosis congenita, hematologic disorder, hereditary disease, hereditary skin disorder, interstitial lung disease, lung disorder, pulmonary fibrosis, respiratory system disorder, skin disorderRecruitingPhase 12026-08-04NCT06817590
A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's DisorderCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Tourette syndrome, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, psychiatric disorder, tic disorderActive, not recruitingPhase 32026-07-22NCT06021522
guideSEQ: Genomic Understanding, Impact, Decision & Ethics in Prenatal SequencingPrenatal Genetic DiagnosisRecruitingNot applicable2026-07-15NCT07610590
Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis ImperfectaBone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfectaActive, not recruitingPhase 32026-05-27NCT05972551
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Myoclonus, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Unverricht-Lundborg syndrome, brain disorder, central nervous system disorder, epilepsy, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, progressive myoclonus epilepsyRecruitingNot stated2026-03-18NCT06593951
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Primary Immunodeficiency Diseases, X-Linked Combined Immunodeficiency Diseases, X-linked disease, hereditary disease, immune system disorder, inborn error of immunity, severe combined immunodeficiencyRecruitingPhase 1 / Phase 22025-12-11NCT03311503
Mycobacteriophage Treatment of Non-tuberculosis MycobacteriaActinomycetales infectious disease, Bacterial Infections and Mycoses, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Infections, Mycobacterium Infections, Nontuberculous, bacterial infectious disease, cystic fibrosis, gram-positive bacterial infections, hereditary disease, lung disorder, mycobacterial infectious disease, pancreas disorder, pulmonary non-tuberculous mycobacterial infection, respiratory system disorderEnrolling by invitationNot stated2025-05-21NCT06262282
Effects of Ketosis on Brain Function in Patients With T1DMAcid-Base Imbalance, Ketosis, Nutritional and Metabolic Diseases, acidosis disorder, metabolic diseaseRecruitingNot applicable2024-12-18NCT04219709
MASA Valve Early Feasibility StudyCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Pulmonary Atresia, Ventricular Outflow Obstruction, aortopulmonary window, cardiovascular disorder, congenital heart disease, heart disorder, heart septal defect, heart valve disorder, persistent truncus arteriosus, pulmonary valve stenosis, pulmonic stenosis, tetralogy of fallot, transposition of the great arteries, vascular malformationRecruitingNot applicable2024-11-06NCT05452720