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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Postoperative and Chronic Pain Genetic Spine and Pectus Surgery StudyBone Diseases, Developmental, Chronic Pain, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kyphosis, Musculoskeletal Abnormalities, Neurologic Manifestations, Pain, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Postoperative Pain, Signs and Symptoms, Spinal Curvatures, bone disorder, musculoskeletal system disorder, pectus excavatum, scoliosis, vertebral column disorderActive, not recruitingNot stated2026-10-08NCT02998138
Validation of Nanosensor Oxygen MeasurementNutritional and Metabolic Diseases, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingPhase 12026-10-07NCT06819683
Setrusumab vs Placebo for 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 2 / Phase 32026-10-06NCT05125809
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, 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 atrophy, spinal muscular atrophy, type IIIRecruitingPhase 22026-10-02NCT07047144
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 DeficiencyCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 1, skin disorder, vascular disorder, vascular hemostatic diseaseActive, not recruitingPhase 12026-09-29NCT05734196
Effect of Patiromer in Children Under 12 Years of Age With High Blood Potassium LevelsHyperkalemia, Nutritional and Metabolic Diseases, Water-Electrolyte Imbalance, metabolic diseaseRecruitingPhase 22026-09-25NCT05766839
Hospital-Based Cluster Trial: Magnetically Controlled Growing Rods Using Distraction IntervalsEarly-Onset Scoliosis Deformity of SpineActive, not recruitingNot applicable2026-09-24NCT04058561
Long-Term Follow-Up Study After Gene-Modified Cell Treatment in Kite StudiesHemic and Lymphatic Diseases, Neoplasms by Site, autoimmune disease, hematologic disorder, hematopoietic and lymphoid system neoplasm, immune system disorder, neoplasmEnrolling by invitationNot stated2026-09-21NCT05041309
Molecular Basis of Food AllergyDigestive System Diseases, Eosinophilia, Hemic and Lymphatic Diseases, Hypersensitivity, Immediate, digestive system disorder, eosinophilic esophagitis, esophageal disorder, esophagitis, food allergy, gastroenteritis, hematologic disorder, immune system disorder, leukocyte disorderEnrolling by invitationNot stated2026-09-21NCT01832324
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet SyndromeEpilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, nervous system disorder, visual epilepsyRecruitingPhase 32026-09-15NCT04462770
A Study to Compare the Long-term Outcomes After Two Different AnaestheticsChemically-Induced Disorders, nervous system disorder, poisoning, toxic encephalopathyActive, not recruitingPhase 32026-09-10NCT03089905
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)autosomal recessive limb-girdle muscular dystrophy type 2IActive, not recruitingPhase 32026-09-09NCT05775848
Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS PatientsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, autoimmune disease, autoimmune lymphoproliferative syndrome, autoimmune lymphoproliferative syndrome type 1, hereditary disease, hypersplenism, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoproliferative syndrome, splenic disorderRecruitingPhase 22026-09-08NCT06730126
Observational Approach Versus Surgical Intervention for StonesCalculi, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Kidney Calculi, Male Urogenital Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Urinary Calculi, Urogenital Diseases, kidney disorder, nephrolithiasis, urinary system disorder, urolithiasisRecruitingNot applicable2026-09-03NCT07718802
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabAtrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscular atrophy, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIActive, not recruitingPhase 32026-09-01NCT05626855
HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)Congenital Hyperinsulinism, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, glucose metabolism disease, hyperinsulinism, hypoglycemia, metabolic disease, pancreas disorderActive, not recruitingPhase 22026-08-26NCT04732416
SAFety and Efficacy of Human Anti-thymocyte ImmunoGlobUlin SAB-142 ARresting Progression of Type 1 DiabetesNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingPhase 22026-08-24NCT07187531
Parental Decision-Making for Children With Relapsed NeuroblastomaDeath, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Pathologic Processes, Pathological Conditions, Signs and Symptoms, epithelial neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingNot stated2026-08-20NCT02282735
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDGDyskinesias, Neurologic Manifestations, PMM2-congenital disorder of glycosylation, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, atactic disorder, non-human animal, nervous system disorderActive, not recruitingPhase 2 / Phase 32026-08-14NCT06892288
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
TReatment for ImmUne Mediated PathopHysiologyBrain Diseases, Metabolic, Digestive System Diseases, Hepatic Insufficiency, Nutritional and Metabolic Diseases, acute liver failure, brain disorder, central nervous system disorder, hepatic encephalopathy, liver disorder, liver failure, metabolic disease, nervous system disorderActive, not recruitingPhase 22026-08-12NCT04862221
A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic EncephalopathyEpileptic EncephalopathyRecruitingPhase 1 / Phase 22026-08-11NCT07227857
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsAbnormalities, Multiple, Barth syndrome, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Cohen syndrome, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Digestive System Diseases, Hemic and Lymphatic Diseases, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, X-linked disease, agranulocytosis, autoimmune disease, bone marrow disorder, cardiovascular disorder, congenital heart disease, exocrine pancreatic insufficiency, heart disorder, hematologic disorder, hereditary disease, immune system disorder, inborn errors of metabolism, leukocyte disorder, leukopenia, lipomatosis, metabolic disease, neutropenia, pancreas disorderRecruitingPhase 32026-08-06NCT06056297
The REPLACE Registry for Cholbam® (Cholic Acid)Abnormalities, Multiple, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Peroxisomal Disorders, Urogenital Diseases, Zellweger spectrum disorders, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of bile acid synthesis, inborn errors of metabolism, kidney disorder, liver disorder, metabolic disease, nervous system disorder, urinary system disorderActive, not recruitingNot stated2026-07-31NCT03115086
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypercalciuria, Hypercalciuric Hypocalcemia, Familial, Hypocalcemia, Male Urogenital Diseases, Musculoskeletal Abnormalities, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urogenital Diseases, Urological Manifestations, Water-Electrolyte Imbalance, calcinosis, calcium metabolic disease, endocrine system disorder, hypoparathyroidism, kidney disorder, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nephrocalcinosis, nephrolithiasis, nervous system disorder, neuromuscular disease, parathyroid gland disorder, urinary system disorder, urolithiasisActive, not recruitingPhase 32026-07-29NCT05680818