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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphatic Abnormalities, Neoplasm, Lymphatic Tissue, Neoplasms by Histologic Type, cystic hygroma, lymphangioma, lymphatic system disorder, neoplasmRecruitingPhase 2 / Phase 32026-10-09NCT05948943
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Sickle Cell Trait, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingPhase 32026-09-28NCT06439082
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's DiseaseCognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Huntington disease, basal ganglia disorder, brain disorder, central nervous system disorder, choreatic disease, cognitive disorder, dementia, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, psychiatric disorderRecruitingPhase 32026-09-22NCT07326709
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease PatientsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Sickle Cell Trait, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseActive, not recruitingPhase 32026-09-14NCT03814746