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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuriaActive, not recruitingNot stated2026-09-03NCT05776472
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR VariantAlzheimer disease, Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cognitive disorder, dementia, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, proteostasis deficiencies, psychiatric disorder, tauopathyRecruitingPhase 32026-08-04NCT06563895
Phase 3, Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants With Early Alzheimer's Disease (Polaris-AD)Alzheimer disease, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyActive, not recruitingPhase 32025-10-02NCT05531526