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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
The AIRTIVITY™ Study: A Study to Find Out Whether BI 1291583 Helps People With Bronchiectasisbronchial disorder, bronchiectasis, respiratory system disorderRecruitingPhase 32026-10-01NCT06872892
A Study to Find Out How Nerandomilast is Tolerated, Handled by the Body, and if it Helps Children and Adolescents With Interstitial Lung Disease (FIBRONEER-chILD)Fibrosing Interstitial Lung DiseaseRecruitingPhase 32026-09-29NCT07366034
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
A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)Chronic Disease, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, chronic renal failure syndrome, kidney disorder, kidney failure, urinary system disorderRecruitingPhase 32026-09-16NCT07107945
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients 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 diseaseRecruitingPhase 32026-07-24NCT05933057
Registry for Patients With X-Linked HypophosphatemiaBone 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 disorderRecruitingNot stated2026-07-09NCT03193476
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular DiseasesAlport syndrome, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Hemorrhage, IgA glomerulonephritis, Male Urogenital Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Urogenital Abnormalities, Urogenital Diseases, autoimmune disease, blood coagulation disease, cardiovascular disorder, connective tissue disorder, focal segmental glomerulosclerosis, glomerulonephritis, hematologic disorder, hemorrhagic disease, hereditary nephritis, immune system disorder, immunoglobulin A vasculitis, kidney disorder, macular corneal dystrophy, minimal change disease, nephritis, nephrosis, purpura, skin disorder, skin vascular disease, type III hypersensitivity disease, urinary system disorder, vascular disorder, vascular hemostatic disease, vasculitisRecruitingPhase 22026-05-12NCT05003986
International Cooperative Treatment Protocol for Children and Adolescents With Lymphoblastic LymphomaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingPhase 32026-05-06NCT04043494
International Study for Treatment of High Risk Childhood Relapsed ALL 2010Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute lymphoblastic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmRecruitingPhase 22026-03-17NCT03590171
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).growth delay due to insulin-like growth factor type 1 deficiencyRecruitingNot stated2026-02-06NCT00903110
A Study of Galcanezumab (LY2951742) in Participants 6 to 17 Years of Age With Episodic MigraineHeadache, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and SymptomsActive, not recruitingPhase 32026-01-26NCT03432286
CytOSorb TreatMent Of Critically Ill PatientS RegistryAcute-On-Chronic Liver Failure, Chemically-Induced Disorders, Digestive System Diseases, Disease Attributes, Drug Misuse, Drug Overdose, Hemic and Lymphatic Diseases, Hepatic Insufficiency, Infarction, Infections, Inflammation, Lymphohistiocytosis, Hemophagocytic, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Postoperative Complications, Postoperative Vasoplegic Syndrome, Prescription Drug Misuse, Respiration Disorders, Rhabdomyolysis, Sepsis, Shock, Shock, Cardiogenic, Systemic Inflammatory Response Syndrome, Vasoplegia, acute liver failure, adult acute respiratory distress syndrome, burn, cardiovascular disorder, cytokine release syndrome, heart disorder, histiocytosis, infectious disease, injury, ischemic disease, liver disorder, liver failure, lung disorder, lymphatic system disorder, muscle tissue disorder, musculoskeletal system disorder, myocardial infarction, myocardial ischemia, non-Langerhans cell histiocytosis, pancreas disorder, pancreatitis, psychiatric disorder, respiratory system disorder, substance-related disorder, toxic shock syndrome, vascular disorderRecruitingNot stated2025-09-11NCT05146336
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years OldCongenital, 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 22025-07-10NCT06769633