Alnylam Pharmaceuticals Inc
ALNYHealthcare|Biotechnology|USA
$298.76
-14.03 (-4.49%)
Tangible Book
$8.06
Graham Number
$32.23
Clinical Trials (95)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT07052903 TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy | Transthyretin Amyloidosis With Cardiomyopathy | P3 | Recruiting | 1,250 | RCT, Double-blind | Nucresiran, Sterile Normal Saline (0.9% NaCl) |
| NCT04153149 HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | P3 | Active | 655 | RCT, Double-blind | Vutrisiran, Sterile Normal Saline (0.9% NaCl) |
| NCT07181109 Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease | High Risk Cardiovascular Disease, Hypertension | P3 | Recruiting | 11,000 | RCT, Double-blind | Zilebesiran, Placebo |
| NCT07223203 TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy | Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN | P3 | Recruiting | 125 | RCT, Open-label | Nucresiran, Vutrisiran |
| NCT07465224 A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus | Type 2 Diabetes Mellitus (T2DM) | P2 | Recruiting | 40 | RCT, Double-blind | ALN-4324, Placebo |
| NCT06225544 Lumasiran in Hyperoxalaemic Patients on Haemodialysis | Haemodialysis, Chronic Kidney Disease Requiring Chronic Dialysis | P2 | Recruiting | 50 | RCT, Double-blind | Lumasiran, 0.9% Sodium Chloride (placebo) |
| NCT07553442 A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran | Hypertension | P2 | Recruiting | 93 | RCT, Double-blind | ALN-AGT01 RVR, Placebo, Zilebesiran |
| NCT06845202 A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM | Obese or Overweight Healthy Volunteers, Type 2 Diabetes Mellitus (T2DM) | P1P2 | Recruiting | 144 | RCT, Double-blind | ALN-4324, Placebo |
| NCT07463846 A Study to Evaluate ALN-2232 in Participants With Obesity | Obesity | P1P2 | Recruiting | 156 | RCT, Double-blind | ALN-2232, Placebo, Tirzepatide |
| NCT06659640 A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT) | Hereditary Hemorrhagic Telangiectasia | P1P2 | Recruiting | 120 | RCT, Double-blind | ALN-6400, Placebo |
| NCT06393712 A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy | Cerebral Amyloid Angiopathy | P2 | Recruiting | 200 | RCT, Double-blind | Placebo, ALN-APP |
| NCT07295717 A Study to Evaluate ALN-4285 in Adult Healthy Volunteers | Healthy Volunteers | P1 | Recruiting | 76 | RCT, Double-blind | ALN-4285, Placebo |
| NCT07214727 A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease | Alzheimer's Disease | P1 | Recruiting | 50 | RCT, Double-blind | ALN-5288, Placebo |
| NCT06585449 A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease | Huntington's Disease | P1 | Recruiting | 66 | RCT, Double-blind | ALN-HTT02, Placebo |
| NCT07535606 A Study to Evaluate ALN-4915 in Adult Healthy Volunteers | Healthy Volunteers | P1 | Recruiting | 44 | RCT, Double-blind | ALN-4915, Placebo |
| NCT05231785 A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD | Early-Onset Alzheimer Disease | P1 | Recruiting | 60 | RCT, Double-blind | ALN-APP, Placebo |
| NCT06600321 A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma | Advanced Hepatocellular Carcinoma, Metastatic Hepatocellular Carcinoma | P1 | Recruiting | 158 | Open-label | ALN-BCAT, Pembrolizumab |
| NCT07624071 A Study to Evaluate ALN-6222 in Participants With Obesity | Obesity | P1 | Recruiting | 88 | RCT, Double-blind | ALN-6222, Placebo |
| NCT06360289 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 Polyneuropathy | Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State | Active | 346 | — | — | |
| NCT05040373 Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program | Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy | Recruiting | 10 | — | — | |
| NCT04883905 ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) | Acute Hepatic Porphyria | Recruiting | 150 | — | — | |
| NCT04982393 BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 | Active | 207 | — | — | |
| NCT07358078 DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting | Transthyretin Amyloidosis With Cardiomyopathy | Recruiting | 2,000 | — | — | |
| NCT05577819 Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF | Transthyretin Amyloidosis, Heart Failure | N/A | Recruiting | 515 | Open-label | — |
| NCT04561518 ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) | Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis | Recruiting | 1,500 | — | — | |
| NCT03681184 A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) | P3 | Completed | 39 | RCT, Double-blind | Placebo, Lumasiran |
| NCT02510261 The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) | Amyloidosis | P3 | Completed | 211 | Open-label | Patisiran |
| NCT03338816 ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) | Acute Hepatic Porphyria, Acute Intermittent Porphyria | P3 | Completed | 94 | RCT, Double-blind | Givosiran, Placebo |
| NCT06679946 A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | P3 | By Invite | 700 | Open-label | Vutrisiran |
| NCT03759379 HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) | Amyloidosis, Hereditary, Transthyretin Amyloidosis | P3 | Completed | 164 | RCT, Open-label | Patisiran, Vutrisiran |
| NCT03997383 APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | P3 | Completed | 360 | RCT, Double-blind | Placebo, Patisiran |
| NCT02319005 ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary | P3 | Completed | 206 | RCT, Double-blind | Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl) |
| NCT03862807 Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant | Amyloidosis, Familial, Transthyretin Amyloidosis | P3 | Completed | 24 | Open-label | Patisiran |
| NCT03905694 A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1) | P3 | Completed | 18 | Open-label | Lumasiran |
| NCT04152200 A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1, Primary Hyperoxaluria | P3 | Completed | 21 | Open-label | Lumasiran |
| NCT01960348 APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis | TTR-mediated Amyloidosis, Amyloidosis, Hereditary | P3 | Completed | 225 | RCT, Double-blind | patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) |
| NCT03672188 Study of VIR-2218 in Healthy Subjects and Patients With Chronic Hepatitis B | Chronic Hepatitis B | P1P2 | Completed | 82 | RCT, Double-blind | VIR-2218, Placebo |
| NCT04412863 Study of VIR-2218 With or Without Pegylated Interferon Alpha-2a for Treatment of Chronic Hepatitis B Virus Infection | Chronic Hepatitis B | P2 | Completed | 84 | Open-label | VIR-2218, pegylated interferon-alfa 2a |
| NCT04507269 Study of VIR-2218 in Patients With Chronic Hepatitis B in Mainland China | Hepatitis B, Chronic | P2 | Completed | 21 | RCT, Double-blind | VIR-2218, Placebo |
| NCT05761301 A Phase 1/2 Study to Evaluate ALN-KHK in Overweight to Obese Healthy Volunteers and Obese Patients With T2DM | Type 2 Diabetes Mellitus (T2DM) | P1P2 | Terminated | 49 | RCT, Double-blind | ALN-KHK, Placebo |
| NCT03303313 A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome | P2 | Withdrawn | — | Open-label | Cemdisiran |
| NCT02706886 Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) | P1P2 | Completed | 52 | RCT, Single-blind | Lumasiran, Placebo |
| NCT06272487 Zilebesiran as Add-on Therapy in Patients With High Cardiovascular Risk and Hypertension Not Adequately Controlled by Standard of Care Antihypertensive Medications (KARDIA-3) | High Cardiovascular Risk, Hypertension | P2 | Completed | 375 | RCT, Double-blind | Zilebesiran, Placebo |
| NCT01617967 Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis | TTR-mediated Amyloidosis | P2 | Completed | 29 | Open-label | Patisiran |
| NCT07575308 HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB | Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB) | P2 | Not Yet Recruiting | 24 | RCT, Double-blind | ALN-6400 |
| NCT02595983 The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant | Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP) | P2 | Completed | 12 | Open-label | Revusiran |
| NCT03841448 A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN) | IgA Nephropathy (IgAN), Berger Disease | P2 | Terminated | 31 | RCT, Double-blind | Placebo, Cemdisiran |
| NCT05256810 A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout | Gout | P1P2 | Terminated | 44 | RCT, Double-blind | ALN-XDH, Placebo |
| NCT00496821 Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial Virus | Respiratory Syncytial Virus Infections | P2 | Completed | 88 | RCT, Double-blind | ALN-RSV01 |
| NCT05161936 A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels | Recurrent Calcium Oxalate Kidney Stone Disease, Elevated Urinary Oxalate Levels | P2 | Terminated | 2 | RCT, Double-blind | Lumasiran, Placebo |
| NCT03767829 A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | P1P2 | Terminated | 32 | RCT, Double-blind | ALN-AAT02, Placebo, ALN-AAT02 |
| NCT01981837 Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | TTR-mediated Amyloidosis | P2 | Completed | 26 | Open-label | ALN-TTRSC (revusiran) for subcutaneous administration |
| NCT03999840 Eculizumab to Cemdisiran Switch in aHUS | Atypical Hemolytic Uremic Syndrome | P2 | Withdrawn | — | RCT, Double-blind | cemdisiran, Placebos |
| NCT04936035 A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate Hypertension | Hypertension | P2 | Completed | 394 | RCT, Double-blind | Placebo, ALN-AGT01 |
| NCT00658086 Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV) | Respiratory Syncytial Virus Infections | P2 | Completed | 24 | RCT, Double-blind | ALN-RSV01, normal saline |
| NCT01961921 The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran) | TTR-mediated Amyloidosis | P2 | Completed | 27 | Open-label | ALN-TTR02 (patisiran) administered by intravenous (IV) infusion |
| NCT02352493 A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH | Paroxysmal Nocturnal Hemoglobinuria (PNH) | P1P2 | Completed | 62 | RCT, Double-blind | ALN-CC5, Sterile Normal Saline (0.9% NaCl) |
| NCT07636811 A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD) | Down Syndrome-Associated Alzheimer's Disease (DS-AD) | P2 | Not Yet Recruiting | 58 | RCT, Double-blind | Mivelsiran, Placebo |
| NCT03350451 An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1 | PH1, Primary Hyperoxaluria | P2 | Completed | 20 | Open-label | Lumasiran |
| NCT06423352 A Study to Evaluate Zilebesiran in Japanese Patients With Mild to Moderate Hypertension | Mild to Moderate Hypertension | P1P2 | Completed | 36 | RCT, Double-blind | Zilebesiran, Placebo |
| NCT02292186 A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | TTR-mediated Amyloidosis | P2 | Completed | 25 | Open-label | Revusiran (ALN-TTRSC) |
| NCT01065935 Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV) | Respiratory Syncytial Virus Infections | P2 | Completed | 87 | RCT, Double-blind | ALN-RSV01, Normal Saline |
| NCT02949830 A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP) | Acute Intermittent Porphyria | P1P2 | Completed | 16 | Open-label | Givosiran |
| NCT05103332 Zilebesiran as Add-on Therapy in Patients With Hypertension Not Adequately Controlled by a Standard of Care Antihypertensive Medication (KARDIA-2) | Hypertension | P2 | Completed | 663 | RCT, Double-blind | Indapamide, Amlodipine, Olmesartan, Placebo, Zilebesiran |
| NCT03505853 A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP) | Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP) | P1 | Completed | 10 | Open-label | Givosiran, 5-probe cocktail |
| NCT02826018 A Study of ALN-HBV in Healthy Adult Volunteers and Non-cirrhotic Patients With Chronic Hepatitis B Virus (HBV) Infection | Hepatitis B, Chronic Hepatitis B | P1 | Terminated | 24 | RCT, Single-blind | ALN-HBV, Sterile Normal Saline (0.9% NaCl) |
| NCT02452372 A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) | Acute Intermittent Porphyria | P1 | Completed | 40 | RCT, Single-blind | givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl) |
| NCT04565717 A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) | Nonalcoholic Steatohepatitis, NASH | P1 | Terminated | 6 | RCT, Double-blind | ALN-HSD, Placebo |
| NCT04601844 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers | Healthy | P1 | Completed | 19 | Open-label | Pozelimab, Cemdisiran |
| NCT02503683 A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease | Antitrypsin Deficiency Liver Disease | P1 | Terminated | 26 | RCT, Single-blind | ALN-AAT, Sterile Normal Saline (0.9% NaCl) |
| NCT01158079 Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment | Solid Tumors | P1 | Completed | 7 | Open-label | ALN-VSP02 |
| NCT01148953 Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis | Transthyretin Mediated Amyloidosis (ATTR) | P1 | Completed | 32 | RCT, Single-blind | ALN-TTR01, Sterile Normal Saline (0.9% NaCl) |
| NCT01437059 Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C) | Elevated LDL-Cholesterol (LDL-C) | P1 | Completed | 32 | RCT, Single-blind | ALN-PCS02, Sterile Normal Saline (0.9% NaCl) |
| NCT02797847 A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects | Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) | P1 | Completed | 80 | RCT, Single-blind | ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl) |
| NCT05661916 A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects | Transthyretin-Mediated Amyloidosis | P1 | Completed | 96 | RCT, Double-blind | ALN-TTRSC04, Placebo |
| NCT02053454 A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers | Transthyretin (TTR)-Mediated Amyloidosis | P1 | Completed | 12 | RCT, Double-blind | patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) |
| NCT01559077 Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects | TTR-mediated Amyloidosis | P1 | Completed | 17 | RCT, Single-blind | ALN-TTR02, Sterile Normal Saline (0.9% NaCl) |
| NCT00882180 Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver Involvement | Solid Tumors | P1 | Completed | 41 | Open-label | ALN-VSP02 |
| NCT03934307 A Study to Evaluate ALN-AGT01 in Patients With Hypertension | Hypertension | P1 | Completed | 124 | RCT, Double-blind | ALN-AGT01, ALN-AGT01-Matching Placebo, Irbesartan, Irbesartan-Matching Placebo |
| NCT01814839 A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers | TTR-mediated Amyloidosis | P1 | Completed | 85 | RCT, Double-blind | ALN-TTRSC (revusiran), Sterile Normal Saline (0.9% NaCl) |
| NCT02314442 A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C) | Hypercholesterolemia | P1 | Completed | 70 | RCT, Single-blind | ALN-PCSSC, Sterile Normal Saline (0.9% NaCl) |
| NCT06675565 A Study to Evaluate ALN-AGT01 RVR in Adult Healthy Volunteers | Healthy Volunteers | P1 | Completed | 42 | RCT, Double-blind | ALN-AGT01 RVR, Placebo |
| NCT05505838 Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy | Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy | NO_LONGER_AVAILABLE | — | — | Patisiran | |
| NCT03774784 A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease | Amyloidosis, Leukocyte Chemotactic Factor 2 Amyloidosis | Terminated | 26 | — | — | |
| NCT03547297 INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP | Acute Hepatic Porphyria, Hepatic Porphyrias | Terminated | 212 | — | — | |
| NCT02252653 DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis | Familial Amyloidotic Cardiomyopathy (FAC) | Completed | 1,010 | — | — | |
| NCT04056481 Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria | Acute Hepatic Porphyria | APPROVED_FOR_MARKETING | — | — | Givosiran | |
| NCT04125472 Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria | APPROVED_FOR_MARKETING | — | — | Lumasiran | |
| NCT03237494 TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects | Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Completed | 5,028 | — | — | |
| NCT04201418 A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation | Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy | Completed | 67 | — | Patisiran | |
| NCT04569903 Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records | Transthyretin Amyloidosis | Early P1 | Withdrawn | — | Open-label | — |
| NCT05940922 RWE-based Treatment Patterns and Outcomes in CIDP | Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barre Syndrome | Unknown | 170 | — | — | |
| NCT02939820 Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR) | TTR-mediated Amyloidosis, Amyloidosis, Hereditary | APPROVED_FOR_MARKETING | — | — | patisiran (ALN-TTR02) | |
| NCT02240784 EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP) | Acute Hepatic Porphyria | Completed | 136 | — | — | |
| NCT05023889 Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy | Polyneuropathies, Wild Type ATTR Amyloidosis | Early P1 | Completed | 10 | Open-label | patisiran |