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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 1 / Phase 22026-10-08NCT05987449
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
X-linked Hypophosphatemia Disease Monitoring ProgramBone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypophosphatemia, Familial, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Rickets, X-Linked Hypophosphatemic, Urogenital Diseases, X-linked hypophosphatemic rickets, bone disorder, calcium metabolic disease, hereditary disease, hypophosphatemia, hypophosphatemic rickets, inborn errors of metabolism, inborn metal metabolism disorder, kidney disorder, metabolic disease, musculoskeletal system disorder, nutritional deficiency disease, nutritional disorder, phosphorus metabolism disease, renal tubular transport disease, rickets, urinary system disorder, vitamin D deficiency, vitamin deficiency disorderActive, not recruitingNot stated2026-08-28NCT03651505
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 32026-08-24NCT05183035
Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative ColitisDigestive System Diseases, colitis, colonic disorder, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorder, ulcerative colitisActive, not recruitingPhase 32026-08-03NCT04624230
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)Noonan syndrome 5, low grade gliomaActive, not recruitingPhase 32026-05-14NCT05566795
Portable Sleep Monitors in Children With Autism Spectrum DisorderApnea, Dyssomnias, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Respiration Disorders, Signs and Symptoms, Sleep Disorders, Intrinsic, autism spectrum disorder, nervous system disorder, neurodevelopmental disorder, obstructive sleep apnea syndrome, pervasive developmental disorder, psychiatric disorder, respiratory system disorder, sleep apnea syndrome, sleep-wake disorderRecruitingNot stated2026-01-26NCT07367022
The IPAD Cohort Study: Insomnia and Positive Airway Pressure Adherence in Children and AdolescentsApnea, Dyssomnias, Respiration Disorders, Sleep Disorders, Intrinsic, insomnia, nervous system disorder, obstructive sleep apnea syndrome, psychiatric disorder, respiratory system disorder, sleep apnea syndrome, sleep-wake disorderRecruitingNot stated2025-09-23NCT07176767
Low-value Care, and Variation in Practice for Children Hospitalized With BronchiolitisInfections, bronchial disorder, bronchiolitis, bronchitis, lung disorder, obstructive lung disease, respiratory system disorder, respiratory tract infectious disorderRecruitingNot stated2025-07-23NCT06506474
Comparing Pain Relief Between Two Methods of Freezing Injections in Children Having Their Appendix RemovedNeurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, agnosia, nervous system disorder, perceptual disordersEnrolling by invitationNot applicable2025-05-16NCT06945263
Global Hip Dysplasia RegistryCongenital Abnormalities, Congenital Dysplasia Of The Hip, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hip Dislocation, Hip Dislocation, Congenital, Hip Dysplasia, Congenital, Nonsyndromic, Hip Injuries, Joint Dislocations, Musculoskeletal Abnormalities, arthropathy, developmental dysplasia of the hip, injury, musculoskeletal system disorderRecruitingNot stated2024-11-18NCT04117685
Visual Telerehabilitation in Children, Adolescents and Young Adults With Hemianopsia Consecutive to a Brain TumourHemianopsia, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, blindness (disorder), eye disorder, nervous system disorder, vision disorderNot yet recruitingNot applicable2024-04-12NCT06362434
SCFE Longitudinal International Prospective RegistryBone Diseases, Developmental, Epiphyses, Slipped, bone disorder, epiphysiolysis of the hip, musculoskeletal system disorder, osteochondrodysplasiaRecruitingNot stated2024-04-05NCT04117841
Measures of Respiratory Health RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Respiration Disorders, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2022-08-15NCT05501587
Canadian Observational Study Evaluating the Long-term IMPACT of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators on People With CFCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Respiration Disorders, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2022-01-20NCT05200429
Imaging Measures of Respiratory Health RegistryRespiration Disorders, respiratory system disorderRecruitingNot stated2021-11-02NCT05102825