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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World SettingNutritional and Metabolic Diseases, amyloidosis, cardiomyopathy, cardiovascular disorder, heart disorder, metabolic disease, proteostasis deficienciesRecruitingNot stated2026-10-09NCT07358078
A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesirancardiovascular disorder, hypertensive disorder, vascular disorderRecruitingPhase 22026-10-08NCT07553442
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With PolyneuropathyNutritional and Metabolic Diseases, amyloidosis, metabolic disease, nervous system disorder, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, proteostasis deficienciesRecruitingPhase 32026-10-08NCT07223203
A Study to Evaluate ALN-5288 in Patients With Alzheimer's DiseaseAlzheimer disease, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyRecruitingPhase 12026-10-08NCT07214727
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance ProgramAmyloid 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, polyneuropathy, proteostasis deficienciesRecruitingNot stated2026-10-08NCT05040373
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)primary hyperoxaluria type 1Active, not recruitingNot stated2026-10-06NCT04982393
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, hepatic porphyria, hereditary coproporphyria, hereditary disease, hereditary skin disorder, liver disorder, metabolic disease, porphyria, porphyria due to ALA dehydratase deficiency, skin disorderRecruitingNot stated2026-10-06NCT04883905
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMBCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Genital Diseases, Hemic and Lymphatic Diseases, Hemorrhage, Menorrhagia, Menstruation Disturbances, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Urogenital Diseases, Uterine Hemorrhage, blood coagulation disease, blood platelet disease, coagulation protein disease, female reproductive system disorder, hematologic disorder, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, uterine disorder, von Willebrand disease (hereditary or acquired)RecruitingPhase 22026-09-18NCT07575308
Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Diseasecardiovascular disorder, hypertensive disorder, vascular disorderRecruitingPhase 32026-09-18NCT07181109
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid AngiopathyNutritional and Metabolic Diseases, amyloidosis, brain disorder, cardiovascular disorder, central nervous system disorder, cerebral amyloid angiopathy, cerebral arterial disease, cerebrovascular disorder, intracranial arterial disease, metabolic disease, nervous system disorder, proteostasis deficiencies, vascular disorderActive, not recruitingPhase 22026-09-18NCT06393712
A Study to Evaluate ALN-4915 in Adult Healthy VolunteersHealthy VolunteersRecruitingPhase 12026-09-16NCT07535606
A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes MellitusNutritional and Metabolic Diseases, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, type 2 diabetes mellitusRecruitingPhase 22026-09-16NCT07465224
A Study to Evaluate ALN-2232 in Participants With ObesityBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nutritional disorder, obesity disorder, overnutritionRecruitingPhase 1 / Phase 22026-09-16NCT07463846
A Study to Evaluate ALN-4285 in Adult Healthy VolunteersHealthy VolunteersRecruitingPhase 12026-09-16NCT07295717
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With CardiomyopathyNutritional and Metabolic Diseases, amyloidosis, cardiomyopathy, cardiovascular disorder, heart disorder, metabolic disease, proteostasis deficienciesRecruitingPhase 32026-09-16NCT07052903
A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DMBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, nutritional disorder, obesity disorder, overnutrition, type 2 diabetes mellitusActive, not recruitingPhase 1 / Phase 22026-09-16NCT06845202
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epistaxis, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, cardiovascular disorder, hematologic disorder, hemorrhagic disease, hereditary hemorrhagic telangiectasia, nasal disorder, otorhinolaryngologic disease, respiratory system disorder, telangiectasis, vascular disorder, vascular hemostatic disease, vascular malformationActive, not recruitingPhase 1 / Phase 22026-09-16NCT06659640
A Study to Evaluate ALN-HTT02 in Adult Patients 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 12026-09-16NCT06585449
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOADAlzheimer disease, brain disorder, central nervous system disorder, cognitive disorder, dementia, nervous system disorder, neurodegenerative disease, psychiatric disorder, tauopathyRecruitingPhase 12026-09-14NCT05231785
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)Amyloid 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, polyneuropathy, proteostasis deficienciesRecruitingNot stated2026-09-08NCT04561518
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Down syndrome, Neurobehavioral Manifestations, Neurologic Manifestations, chromosomal disorder, hereditary disease, intellectual disability, nervous system disorderRecruitingPhase 22026-09-04NCT07636811
A Study to Evaluate ALN-6222 in Participants With ObesityBody Weight, Nutritional and Metabolic Diseases, Overweight, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nutritional disorder, obesity disorder, overnutritionRecruitingPhase 12026-09-03NCT07624071
Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEFNutritional and Metabolic Diseases, amyloidosis, cardiovascular disorder, diastolic heart failure, familial amyloid neuropathy, heart disorder, heart failure, metabolic disease, proteostasis deficienciesRecruitingNot applicable2026-09-01NCT05577819
Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathyfamilial amyloid neuropathyActive, not recruitingNot stated2026-08-12NCT06360289
A Study to Evaluate ALN-BCAT in Patients With 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 12026-06-17NCT06600321