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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 22026-10-06NCT03962543
Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingPhase 42026-07-08NCT05548283
Utility of PCD Diagnostics to Improve Clinical CareAbnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, hereditary disease, otorhinolaryngologic disease, primary ciliary dyskinesia, respiratory system disorderRecruitingNot applicable2026-07-06NCT03704207
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic MalformationsCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphatic Abnormalities, Neoplasm, Lymphatic Tissue, Neoplasms by Histologic Type, cardiovascular disorder, lymphangioma, lymphatic system disorder, microcystic lymphatic malformation, neoplasm, vascular malformationActive, not recruitingPhase 32026-03-27NCT06239480
Pulmonary Hypertension Association Registrycardiovascular disorder, chronic thromboembolic pulmonary hypertension, hypertensive disorder, lung disorder, pulmonary arterial hypertension, pulmonary hypertension, respiratory system disorder, vascular disorderRecruitingNot stated2025-11-14NCT04071327
Prospective Study of Pregnancy in Women With Cystic FibrosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-11-03NCT04828382
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical OutcomesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingNot stated2025-10-10NCT03350828