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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and OlderCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderEnrolling by invitationPhase 32026-04-29NCT05844449
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell diseaseRecruitingNot stated2026-03-05NCT06923111
FaR-RMS: An Overarching Study for Children and Adults With Frontline and Relapsed RhabdoMyoSarcomaMyosarcoma, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, myomatous neoplasm, neoplasm, rhabdomyosarcoma, sarcomaRecruitingPhase 1 / Phase 22024-05-23NCT04625907