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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, restrictive cardiomyopathy, subvalvular aortic stenosisActive, not recruitingNot stated2026-09-02NCT05112237
Study of Lurbinectedin in Combination With Doxorubicin Versus Doxorubicin Alone as First-line Treatment in Participants With Metastatic Leiomyosarcoma (SaLuDo)Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, leiomyosarcoma, myomatous neoplasm, neoplasm, sarcomaActive, not recruitingPhase 32026-06-18NCT06088290
A Study of TTI-101 as Monotherapy and in Combination in Participants With Locally Advanced or Metastatic, and Unresectable Hepatocellular CarcinomaDigestive System Diseases, Digestive System Neoplasms, Liver Neoplasms, Neoplasms by Histologic Type, Neoplasms by Site, adenocarcinoma, carcinoma, epithelial neoplasm, hepatocellular carcinoma, liver disorder, neoplasmRecruitingPhase 1 / Phase 22026-04-27NCT05440708
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)Nutritional and Metabolic Diseases, amyloidosis, metabolic disease, proteostasis deficienciesEnrolling by invitationPhase 32026-04-17NCT05667493
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR ParticipantsAmyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, familial amyloid neuropathy, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficienciesActive, not recruitingPhase 32025-10-14NCT04988386