Ionis Pharmaceuticals Inc
IONSHealthcare|Biotechnology|USA
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Clinical Trials (156)
| Trial | Conditions | Phase | Status | Enrollment | Design | Drug |
|---|---|---|---|---|---|---|
| NCT04136171 CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | P3 | Active | 1,438 | RCT, Double-blind | Eplontersen, Placebo |
| NCT05071300 A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | P3 | Active | 151 | Open-label | Eplontersen |
| NCT05681351 CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG) | Severe Hypertriglyceridemia | P3 | Active | 885 | Open-label | Olezarsen |
| NCT04849741 A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | Alexander Disease | P3 | Active | 54 | RCT, Double-blind | zilganersen, Placebo |
| NCT05130450 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) | Familial Chylomicronemia Syndrome | P3 | Active | 60 | Open-label | Olezarsen |
| NCT04768972 FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS) | Amyotrophic Lateral Sclerosis | P3 | Active | 89 | RCT, Double-blind | ION363, Placebo |
| NCT05185843 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen | Familial Chylomicronemia Syndrome | P3 | Active | 24 | Open-label | Olezarsen |
| NCT07298447 Donidalorsen Treatment in Children With Hereditary Angioedema | Hereditary Angioedema (HAE) | P3 | Recruiting | 20 | Open-label | Donidalorsen |
| NCT05392114 A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) | Hereditary Angioedema | P3 | Active | 154 | Open-label | Donidalorsen |
| NCT06914609 REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome | Angelman Syndrome | P3 | Recruiting | 158 | RCT, Double-blind | ION582, Placebo |
| NCT06430385 ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | P1P2 | Recruiting | 48 | RCT, Double-blind | ION440 |
| NCT07531745 ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome | Dravet Syndrome | P1P2 | Recruiting | 32 | Open-label | ION337 |
| NCT05127226 HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome | Angelman Syndrome | P1P2 | Recruiting | 70 | Open-label | ION582 |
| NCT07604974 A Study to Assess the Safety, Tolerability and Efficacy of ION775 | Hypertriglyceridemia, Severe Hypertriglyceridemia | P2 | Recruiting | 72 | RCT, Open-label | ION775 |
| NCT06153966 PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 | Prion Disease | P1P2 | Recruiting | 76 | RCT, Double-blind | ION717, Placebo |
| NCT04165486 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) | Multiple System Atrophy | P1 | Recruiting | 40 | RCT, Double-blind | ION464, Placebo |
| NCT06150716 Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) | Pelizaeus-Merzbacher Disease | P1 | Recruiting | 24 | RCT, Open-label | ION356 |
| NCT05659901 Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease | Pelizaeus-Merzbacher Disease | Recruiting | 32 | — | — | |
| NCT06073574 CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Active | 150 | — | — | |
| NCT02714764 Evaluation of Outcome Metrics in Alexander Disease | Alexander Disease | Recruiting | 200 | — | — | |
| NCT03047369 The Myelin Disorders Biorepository Project | Leukodystrophy, White Matter Disease | Recruiting | 12,000 | — | — | |
| NCT06073587 The CARDIO-TTRansform Scintigraphy Sub-study | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Active | 150 | — | — | |
| NCT00607373 Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia | Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant | P3 | Completed | 51 | RCT, Double-blind | mipomersen, Placebo |
| NCT00694109 An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia | Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant | P3 | Completed | 144 | Open-label | Mipomersen Sodium |
| NCT04136184 NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | P3 | Completed | 168 | RCT, Open-label | Inotersen, Eplontersen |
| NCT05610280 A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia | Hypertriglyceridemia, Cardiovascular Diseases | P3 | Completed | 1,478 | RCT, Double-blind | Olezarsen, Placebo |
| NCT05079919 A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia | Severe Hypertriglyceridemia | P3 | Completed | 617 | RCT, Double-blind | Olezarsen, Placebo |
| NCT02658175 The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome | Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | P3 | Completed | 68 | Open-label | Volanesorsen |
| NCT03070119 Long-Term Evaluation of BIIB067 (Tofersen) | ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation | P3 | Completed | 139 | Open-label | Tofersen |
| NCT02623699 An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis | P3 | Completed | 176 | RCT, Double-blind | Tofersen, Placebo |
| NCT00017407 Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung Cancer | Lung Cancer | P3 | Completed | — | — | ISIS 3521, carboplatin, paclitaxel |
| NCT02175004 Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) | FAP, Familial Amyloid Polyneuropathy | P3 | Completed | 135 | Open-label | Inotersen |
| NCT00794664 Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who Are Not on Apheresis | Hypercholesterolemia, Coronary Heart Disease | P3 | Completed | 58 | RCT, Double-blind | Mipomersen, Placebo |
| NCT00770146 Safety and Efficacy of Mipomersen (ISIS 301012) As Add-on Therapy in High Risk Hypercholesterolemic Patients | Hypercholesterolemia, Coronary Heart Disease | P3 | Completed | 158 | RCT, Double-blind | Mipomersen sodium, Placebo |
| NCT05667493 An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | P3 | By Invite | 1,400 | Open-label | Eplontersen |
| NCT05139810 OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) | Hereditary Angioedema | P3 | Completed | 91 | RCT, Double-blind | Donidalorsen, Placebo |
| NCT00048295 Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease | Crohn's Disease | P3 | Completed | 150 | RCT, Double-blind | Alicaforsen |
| NCT00706849 Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease | Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease | P3 | Completed | 124 | RCT, Double-blind | mipomersen sodium, placebo |
| NCT01737398 Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy | FAP, Familial Amyloid Polyneuropathy | P2P3 | Completed | 173 | RCT, Double-blind | Inotersen, Placebo |
| NCT02527343 The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy | Familial Partial Lipodystrophy | P2P3 | Terminated | 40 | RCT, Double-blind | volanesorsen, Placebo |
| NCT02300233 The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia | Hypertriglyceridemia | P3 | Completed | 114 | RCT, Double-blind | Volanesorsen, Placebo |
| NCT04568434 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) | Familial Chylomicronemia Syndrome | P3 | Completed | 66 | RCT, Double-blind | Olezarsen, Placebo |
| NCT05552326 A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia | Severe Hypertriglyceridemia | P3 | Completed | 446 | RCT, Double-blind | Olezarsen, Placebo |
| NCT00048113 Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease | Crohn's Disease | P3 | Completed | 150 | RCT, Double-blind | Alicaforsen |
| NCT02211209 The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome | Familial Chylomicronemia Syndrome | P3 | Completed | 67 | RCT, Double-blind | Volanesorsen, Placebo |
| NCT03446144 Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD) | Geographic Atrophy, Age Related Macular Degeneration | P2 | Withdrawn | — | RCT, Double-blind | IONIS-FB-Lrx, Placebo (sterline saline 0.9%) |
| NCT00231569 Dose-escalating Safety Study in Subjects on Stable Statin Therapy | Hypercholesterolemia | P2 | Completed | 74 | RCT, Double-blind | ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo |
| NCT01234025 Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC) | Castrate-Resistant Prostate Cancer | P1P2 | Completed | 113 | RCT, Open-label | ISIS EIF4E Rx, ISIS EIF4E Rx, ISIS EIF4E Rx, Prednisone, Docetaxel |
| NCT05143957 A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera | Phlebotomy Dependent Polycythemia Vera | P2 | Completed | 50 | RCT, Open-label | sapablursen |
| NCT00002356 The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the Eyes | Cytomegalovirus Retinitis, HIV Infections | P2 | Completed | — | Open-label | Fomivirsen sodium |
| NCT00477594 Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia | Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant | P2 | Completed | 21 | RCT, Open-label | mipomersen sodium |
| NCT04836182 A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction | Chronic Heart Failure With Reduced Ejection Fraction | P2 | Completed | 72 | RCT, Double-blind | IONIS-AGT-LRx, Placebo |
| NCT04549922 Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19 | Covid19 | P2 | Completed | 111 | RCT, Double-blind | ISIS 721744, Normal Saline |
| NCT03070782 Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease | Elevated Lipoprotein(a), Cardiovascular Disease | P2 | Completed | 286 | RCT, Double-blind | ISIS 681257, Placebo |
| NCT02627820 The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart | Amyloidosis | P2 | Withdrawn | — | Open-label | Isis 420915/GSK 299872 |
| NCT04714320 A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure | Hypertension | P2 | Completed | 160 | RCT, Open-label | Placebo, IONIS-AGT-LRx |
| NCT03360747 Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) | Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | P2 | Completed | 3 | Open-label | AKCEA-ANGPTL3-LRx |
| NCT03714776 A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood Pressure | Mild Hypertension | P2 | Completed | 25 | RCT, Double-blind | Placebo, ISIS 757456 |
| NCT03514420 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL) | Familial Partial Lipodystrophy | P2 | Completed | 4 | Open-label | AKCEA-ANGPTL3-LRx |
| NCT00330330 Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Who Have Not Received Prior Treatment | Type 2 Diabetes Mellitus | P2 | Completed | 96 | RCT, Double-blind | ISIS 113715 |
| NCT02583919 Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 Diabetes | Type 2 Diabetes | P2 | Completed | 79 | RCT, Double-blind | ISIS-GCGRRx- Dose Level 1, ISIS-GCGRRx- Dose Level 2, Placebo |
| NCT00330200 Effects of ISIS 113715 on Insulin Sensitivity, Glucose, and Lipid Metabolism and Energy Expenditure in Type 2 Diabetics | Type 2 Diabetes Mellitus | P2 | Terminated | 2 | RCT, Double-blind | ISIS 113715 |
| NCT03815825 GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD) | Macular Degeneration, Geographic Atrophy | P2 | Completed | 332 | RCT, Double-blind | IONIS-FB-LRx, Placebo |
| NCT05355402 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia | Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease | P2 | Completed | 154 | RCT, Double-blind | Olezarsen, Placebo |
| NCT02553889 A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on Hemodialysis | End-stage Renal Disease (ESRD) | P2 | Completed | 49 | RCT, Double-blind | ISIS 416858, Placebo |
| NCT02824003 Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 Diabetes | Type 2 Diabetes | P2 | Completed | 15 | RCT, Double-blind | ISIS-GCGRRx, Placebo |
| NCT02981602 Safety, Tolerability, Pharmacokinetics and Antiviral Activity of IONIS-HBVRx in Treatment-Naïve Patients With Chronic HBV Infection | Hepatitis B, Chronic Hepatitis B Atypical | P2 | Completed | 31 | RCT, Double-blind | IONIS-HBVRx, Placebo |
| NCT03728634 Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ). | Healthy Volunteers, hATTR Amyloidosis | P1P2 | Completed | 47 | RCT, Double-blind | ION-682884, Placebo |
| NCT03371355 Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease | NAFLD, Diabetes Mellitus, Type 2 | P2 | Completed | 105 | RCT, Double-blind | Placebo, ISIS 703802 40 mg, ISIS 703802 80 mg, ISIS 703802 20 mg |
| NCT04522180 A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly | Acromegaly | P2 | Completed | 34 | RCT, Open-label | GHR-LRX |
| NCT00035945 ISIS 14803-CS2, Treatment With ISIS 14803, Administered IV in Patients With Chronic Hepatitis C Virus Infections | Hepatitis C, Chronic | P2 | Completed | — | — | ISIS 14803 |
| NCT00707746 Safety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects | Metabolic Diseases, Hyperlipidemias | P2 | Completed | 34 | RCT, Double-blind | mipomersen, placebo |
| NCT00048321 ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis | Rheumatoid Arthritis | P2 | Completed | 160 | RCT, Double-blind | ISIS 104838 |
| NCT01234038 Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel | Non-small Cell Lung Cancer | P1P2 | Completed | 116 | RCT, Open-label | ISIS EIF4E Rx, ISIS EIF4E Rx, ISIS EIF4E Rx, Paclitaxel, Carboplatin |
| NCT02160899 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a) | Elevated Lipoprotein(a) | P2 | Completed | 64 | RCT, Double-blind | ISIS-APO(a)Rx, Placebo |
| NCT01414101 Safety, Tolerability, and Pharmacodynamic Study of ISIS CRP Rx in Rheumatoid Arthritis | Rheumatoid Arthritis | P2 | Completed | 51 | RCT, Double-blind | ISIS CRP Rx or Placebo |
| NCT00062816 Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Responding Adequately to the Two Drugs | Hepatitis C, Chronic | P1P2 | Completed | 22 | — | ISIS 14803, peginterferon alfa, ribavirin |
| NCT02476019 A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese Patients | Obesity | P2 | Completed | 13 | RCT, Double-blind | ISIS-FGFR4RX, Placebo |
| NCT00280995 Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | Hypercholesterolemia, Familial | P2 | Completed | 12 | RCT, Open-label | ISIS 301012, ISIS 301012, ISIS 301012, ISIS 301012 |
| NCT04307381 An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema | Hereditary Angioedema | P2 | Completed | 20 | Open-label | Donidalorsen |
| NCT03455777 Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | P2 | Withdrawn | — | Open-label | AKCEA-ANGPTL3-LRX |
| NCT03358030 A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemodialysis | End-stage Renal Disease (ESRD) | P2 | Completed | 213 | RCT, Double-blind | ISIS 416858, Placebo |
| NCT01710852 Antiarrhythmic and Symptomatic Effect of ISIS CRP Rx Targeting CRP in Paroxysmal Atrial Fibrillation | Paroxysmal Atrial Fibrillation | P2 | Completed | 7 | RCT, Double-blind | ISIS CRP Rx, Placebo |
| NCT00063830 ISIS 2302-CS27, A 6-Week, Placebo-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS 2302) in Patients With Mild to Moderate Active Ulcerative Colitis. | Ulcerative Colitis | P2 | Completed | — | — | Alicaforsen |
| NCT04932512 A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224 | Steatohepatitis, Nonalcoholic | P2 | Completed | 160 | RCT, Double-blind | ION224 |
| NCT04083222 A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx | Hypertension | P2 | Completed | 26 | RCT, Double-blind | Placebo, ISIS 757456 |
| NCT04059406 Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) | Beta Thalassemia Intermedia | P2 | Terminated | 29 | RCT, Open-label | sapablursen |
| NCT00002156 A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR ) | Cytomegalovirus Retinitis, HIV Infections | P2 | Completed | 194 | Open-label | Fomivirsen sodium, Ganciclovir |
| NCT01713361 Active Comparator-Controlled Study to Assess Safety and Efficacy of ISIS-FXIRx in Total Knee Arthroplasty | Venous Thromboembolism | P2 | Completed | 315 | RCT, Open-label | ISIS-FXIRx Dose #2, ISIS-FXIRx Dose #3, Enoxaparin |
| NCT04441788 A Study to Assess the Safety, Tolerability, and Efficacy of ION-827359 in Participants With Mild to Moderate Chronic Obstructive Pulmonary Disease (COPD) With Chronic Bronchitis (CB) | Chronic Bronchitis, Chronic Obstructive Pulmonary Disease | P2 | Terminated | 60 | RCT, Double-blind | ION-827359, Placebo, ION-827359 |
| NCT00281008 Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | Hypercholesterolemia, Familial | P2 | Completed | 44 | RCT, Double-blind | ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo |
| NCT00005594 ISIS 2503 in Treating Patients With Advanced Cancer of the Pancreas | Pancreatic Cancer | P2 | Completed | 4 | Open-label | ISIS 2503 |
| NCT00362180 Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration | Lipid Metabolism, Inborn Errors, Hyperlipidemias | P2 | Completed | 38 | RCT, Double-blind | mipomersen, Placebo |
| NCT05314439 A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension | Hypertension | P2 | Completed | 48 | RCT, Double-blind | ION904, Placebo |
| NCT03334214 Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes | Hepatic Steatosis | P2 | Completed | 44 | RCT, Double-blind | IONIS DGAT2Rx, Placebo |
| NCT04014335 A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy | Primary IgA Nephropathy | P2 | Completed | 23 | Open-label | IONIS-FB-LRx |
| NCT02312011 A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 | Myotonic Dystrophy Type 1 | P1P2 | Completed | 48 | RCT, Double-blind | IONIS-DMPKRx, Placebo |
| NCT03548415 Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands | Acromegaly | P2 | Completed | 43 | RCT, Double-blind | IONIS-GHR-LRx, Placebo |
| NCT00063414 ISIS 2302-CS22, A 6-Week, Active-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS 2302) in Patients With Mild to Moderate Active Ulcerative Colitis | Ulcerative Colitis | P2 | Completed | — | — | Alicaforsen |
| NCT03385239 Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD) | Hypertriglyceridemia, Cardiovascular Diseases | P2 | Completed | 114 | RCT, Double-blind | ISIS 678354, Placebo |
| NCT03967249 Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands | Acromegaly | P2 | Completed | 39 | Open-label | IONIS GHR-LRx, Somatostatin Receptor Ligand (SRL) |
| NCT04030598 A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema | Hereditary Angioedema | P2 | Completed | 23 | RCT, Double-blind | Donidalorsen, Placebo |
| NCT01918865 Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 Diabetes | Type 2 Diabetes Mellitus, Obese | P2 | Completed | 92 | RCT, Double-blind | ISIS-PTP1BRx, Placebo, daily OAD (metformin and/or sulfonylurea) |
| NCT01563302 Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced Cancers | Advanced Cancers, DLBCL | P1P2 | Completed | 64 | Open-label | IONIS-STAT3Rx |
| NCT00327626 Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Being Treated With Sulfonylurea | Type 2 Diabetes Mellitus | P2 | Withdrawn | — | RCT, Double-blind | ISIS 113715 |
| NCT01968265 Safety, Tolerability and Efficacy of ISIS-GCCRRx in Type 2 Diabetes | Type 2 Diabetes Mellitus | P2 | Completed | 38 | RCT, Double-blind | ISIS-GCCRRx, Placebo |
| NCT01529424 Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in Hypertriglyceridemia | Hypertriglyceridemia | P2 | Completed | 89 | RCT, Double-blind | ISIS apoC-III Rx, ISIS apoC-III Rx, ISIS apoC-III Rx, Placebo, Placebo, Placebo |
| NCT01647308 Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 Diabetes | Type 2 Diabetes | P2 | Terminated | 15 | RCT, Double-blind | ISIS-APOCIIIRX, Placebo |
| NCT02519036 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease | Huntington's Disease | P1P2 | Completed | 46 | RCT, Double-blind | ISIS 443139 10 mg, ISIS 443139 30 mg, ISIS 443139 60 mg, ISIS 443139 90 mg, ISIS 443139 120 mg |
| NCT00216463 Safety and Tolerability of Varying Load and Dose of ISIS 301012 in People With Elevated LDL-cholesterol Levels | Hypercholesterolemia | P2 | Completed | 50 | RCT, Double-blind | ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo, ISIS 301012 or Placebo |
| NCT01885260 Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 Diabetes | Type 2 Diabetes Mellitus | P2 | Completed | 77 | RCT, Double-blind | ISIS-GCGRRx - Dose Level 1, ISIS-GCGRRx - Dose Level 2, Placebo |
| NCT03108469 Efficacy and Safety of IONIS-PKKRx for Preventive Treatment of Chronic Migraine | Chronic Migraine | P2 | Completed | 30 | RCT, Double-blind | IONIS-PKKRx (ISIS 546254) |
| NCT00455598 Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With Sulfonylurea | Type 2 Diabetes Mellitus | P2 | Completed | 88 | RCT, Double-blind | ISIS 113715 |
| NCT01839604 A Phase I/Ib Study of AZD9150 (ISIS-STAT3Rx) in Patients With Advanced/Metastatic Hepatocellular Carcinoma | Advanced Adult Hepatocellular Carcinoma, Hepatocellular Carcinoma Metastatic | P1 | Completed | 58 | Open-label | AZD9150 |
| NCT03186989 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease | Mild Alzheimer's Disease | P1 | Completed | 46 | RCT, Double-blind | IONIS MAPTRx |
| NCT00519727 Safety Study of ISIS 325568 in Healthy Volunteers | Type 2 Diabetes Mellitus | P1 | Completed | 60 | RCT, Double-blind | ISIS 325568 |
| NCT02709850 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia | Hypertriglyceridemia, Familial Hypercholesterolemia | P1 | Completed | 48 | RCT, Double-blind | IONIS ANGPTL3-LRx, Placebo |
| NCT03263507 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers | Healthy Volunteers | P1 | Completed | 32 | RCT, Double-blind | Placebo, Donidalorsen |
| NCT04731623 A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904 | Healthy Volunteers | P1 | Completed | 40 | RCT, Double-blind | ION904, Placebo |
| NCT03582462 A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy Volunteers | Healthy Participants | P1 | Completed | 66 | RCT, Double-blind | IONIS FXI-LRx, Placebo |
| NCT01061814 A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers | Healthy Volunteer | P1 | Completed | 84 | RCT, Double-blind | mipomersen, Placebo |
| NCT00365781 Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715 | Type 2 Diabetes Mellitus | P1 | Completed | 10 | Open-label | ISIS 113715 |
| NCT01299298 A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers | Healthy Volunteer | P1 | Completed | 20 | RCT, Double-blind | mipomersen, placebo |
| NCT06673069 Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD) | Alzheimer Disease, Down Syndrome | P1 | Terminated | 1 | RCT, Double-blind | ION269 |
| NCT04934891 A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547 | Healthy Volunteers | P1 | Completed | 48 | RCT, Double-blind | ION547, Placebo |
| NCT04659096 A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors | Advanced Solid Tumors | P1 | Completed | 15 | Open-label | ION537 |
| NCT01133366 A Study to Determine the Effects of Multiple Doses of Mipomersen (200 mg SC) on the Pharmacodynamics and Pharmacokinetics of Single-dose Warfarin | Healthy | P1 | Completed | 18 | Open-label | warfarin sodium, mipomersen sodium; warfarin sodium |
| NCT00734240 Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers | Inflammatory Diseases | P1 | Completed | 103 | RCT, Double-blind | ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512, ISIS 353512 |
| NCT01090661 A Study to Evaluate the Effect of Mipomersen on Cardiac Repolarization Conducted in Healthy Subjects | Healthy | P1 | Completed | 60 | RCT, Double-blind | mipomersen sodium, mipomersen sodium, moxifloxacin hydrochloride (Avelox®), placebo, placebo |
| NCT03165864 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers | Thalassemia | P1 | Completed | 36 | RCT, Double-blind | IONIS TMPRSS6-Lrx |
| NCT01041222 Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations | Familial Amyotrophic Lateral Sclerosis | P1 | Completed | 33 | RCT, Double-blind | ISIS 333611 |
| NCT02414594 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a) | Elevated Lipoprotein(a) | P1 | Completed | 60 | RCT, Double-blind | IONIS-APO(a)-LRx, Sterile Normal Saline (0.9% NaCl) |
| NCT05337878 A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants | Healthy Participants | P1 | Completed | 29 | RCT, Double-blind | Placebo, Pelacarsen |
| NCT05579860 A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants | Healthy Participants | P1 | Completed | 104 | RCT, Open-label | Olezarsen |
| NCT03506854 Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients | Renal Impairment | P1 | Completed | 16 | Open-label | ISIS 681257 |
| NCT03101878 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers | Healthy Volunteers | P1 | Completed | 62 | RCT, Double-blind | Ionis AGT-LRx, Placebo |
| NCT02910635 A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers | Abnormalities, Cardiovascular | P1 | Completed | 52 | RCT, Double-blind | Volanesorsen, Moxifloxacin, Placebo |
| NCT03976349 A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease | Parkinson's Disease | P1 | Completed | 62 | RCT, Double-blind | BIIB094, Placebo |
| NCT04398485 A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma | Relapsed Multiple Myeloma, Refractory Multiple Myeloma | P1 | Completed | 23 | Open-label | ION251 |
| NCT04302064 A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants | Healthy Participants | P1 | Completed | 24 | RCT, Double-blind | Eplontersen, Placebo |
| NCT03647228 A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis | Healthy Subjects, Cystic Fibrosis | P1 | Completed | 98 | RCT, Double-blind | IONIS-ENaCRx, Placebo |
| NCT02900027 Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides | Elevated Triglycerides (TG) | P1 | Completed | 56 | RCT, Double-blind | APOC-III-L-Rx, Placebo Comparator |
| NCT00836225 Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers | Type 2 Diabetes Mellitus | P1 | Completed | 103 | RCT, Double-blind | ISIS 388626 |
| NCT01414881 Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects | Healthy Volunteer | P1 | Completed | 20 | Single-blind | mipomersen, Placebo |
| NCT06360237 Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) | Familial Chylomicronemia Syndrome | APPROVED_FOR_MARKETING | — | — | Olezarsen | |
| NCT04288128 Integrated Functional Evaluation of the Cerebellum | Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 7 | Completed | 40 | — | — | |
| NCT00002187 A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus Retinitis | Cytomegalovirus Retinitis, HIV Infections | N/A | Completed | — | — | Fomivirsen sodium |
| NCT00002355 A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the Eyes | Cytomegalovirus Retinitis, HIV Infections | N/A | Completed | 60 | — | Fomivirsen sodium |
| NCT05311488 Early Detection of Neuropathy in ATTRv | Hereditary Amyloidosis, Transthyretin-Related | Completed | 47 | — | — | |
| NCT05259072 carDIo-ttranSSfOrm nucLEar Imaging Study | Amyloidosis, Transthyretin Amyloid Cardiomyopathy | Terminated | 4 | — | — | |
| NCT03400098 ATTR Expanded Access Program (EAP) by Ionis | Amyloidosis, Hereditary | APPROVED_FOR_MARKETING | — | — | Inotersen | |
| NCT03876522 Natural History and Functional Status Study of Patients With Lafora Disease | Lafora Disease | Completed | 33 | — | — | |
| NCT05637697 Angelman Syndrome Video Assessment (ASVA) Source Material Study | Angelman Syndrome | Completed | 73 | — | — | |
| NCT06014541 Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Terminated | 29 | — | — | |
| NCT07487389 Zilganersen Expanded Access Program for Individuals With Alexander Disease | Alexander Disease | AVAILABLE | — | — | zilganersen | |
| NCT06415448 Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema | Hereditary Angioedema | AVAILABLE | — | — | Donidalorsen |