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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination TherapyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 32026-10-09NCT05444257
A ELN-Multicenter Study on Phenotypic Evolution and Clinical OutcomesHemic and Lymphatic Diseases, Myeloproliferative Disorders, Neoplasms by Site, acquired polycythemia vera, blood platelet disease, bone marrow disorder, bone marrow neoplasm, hematologic disorder, hematopoietic and lymphoid system neoplasm, neoplasm, thrombocytosis diseaseRecruitingNot stated2026-07-09NCT07203768
Ultra-high-caloric, Fatty Diet in ALSNutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorderRecruitingNot applicable2026-05-01NCT06280079
Non-ischemic Preservation of the Donor Heart in Heart TransplantationHeart TransplantationActive, not recruitingNot applicable2026-04-21NCT03991923
Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulatorsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderRecruitingNot stated2026-02-24NCT04732910
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia BCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingNot stated2026-02-19NCT06008938
Immunotherapy Combined With Y-90 SIRT Therapy in Advanced Stage Intrahepatic Biliary Tract Cancer (BTC)Neoplasms by Histologic Type, adenocarcinoma, carcinoma, cholangiocarcinoma, epithelial neoplasm, intrahepatic cholangiocarcinoma, neoplasmActive, not recruitingPhase 22025-12-05NCT04238637
Liver Transplantation in Patients With CirrHosis and Severe Acute-on-Chronic Liver Failure: iNdications and outComEsAcute-On-Chronic Liver Failure, Digestive System Diseases, Fibrosis, Hepatic Insufficiency, Pathologic Processes, Pathological Conditions, Signs and Symptoms, acute liver failure, cirrhosis of liver, liver disorder, liver failureRecruitingNot stated2025-10-20NCT04613921
Study of Navtemadlin add-on to Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to RuxolitinibHemic and Lymphatic Diseases, Myeloproliferative Disorders, bone marrow disorder, hematologic disorder, primary myelofibrosisRecruitingPhase 32025-09-25NCT06479135
PIECES - Towards Large-Scale Adaptation and Tailored Implementation of Primary Cancer Prevention ProgramsFocus of Study: Tailored ImplementationEnrolling by invitationNot applicable2025-04-09NCT06718322
INFORM2 Study Uses Nivolumab and Entinostat in Children and Adolescents With High-risk Refractory MalignanciesNeoplasms by Site, central nervous system cancer, central nervous system neoplasm, neoplasm, nervous system disorderRecruitingPhase 1 / Phase 22024-02-07NCT03838042