Ionis Pharmaceuticals, Inc.: all 133 studies

Page 1 of 2. Open to participants first (11), then studies with results posted, then the rest; newest registration first within each.

• CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)RecruitingNCT07782827Phase 3
• A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsRecruitingNCT07727538Phase 3
• A Study to Assess the Safety, Tolerability and Efficacy of ION775RecruitingNCT07604974Phase 2
• ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeRecruitingNCT07531745Phase 1/2
• Donidalorsen Treatment in Children With Hereditary AngioedemaRecruitingNCT07298447Phase 3
• 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)RecruitingNCT06430385Phase 1/2
• Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)RecruitingNCT06150716Phase 1
• An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)Enrolling by invitationNCT05667493Phase 3
• Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseRecruitingNCT05659901
• TEGSEDI Pregnancy Surveillance ProgramNot yet recruitingNCT04270058
• Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)RecruitingNCT04165486Phase 1
• 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 HypertriglyceridemiaCompletedNCT05355402Phase 2Results posted
• OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)CompletedNCT05139810Phase 3Results posted
• A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood PressureCompletedNCT04714320Phase 2Results posted
• A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)CompletedNCT04568434Phase 3Results posted
• A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyCompletedNCT04522180Phase 2Results posted
• 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)TerminatedNCT04441788Phase 2Results posted
• An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary AngioedemaCompletedNCT04307381Phase 2Results posted
• A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PNTerminatedNCT04306510Phase 4Results posted
• 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 PolyneuropathyCompletedNCT04136184Phase 3Results posted
• A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRxCompletedNCT04083222Phase 2Results posted
• Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)TerminatedNCT04059406Phase 2Results posted
• A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary AngioedemaCompletedNCT04030598Phase 2Results posted
• 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 ).CompletedNCT03728634Phase 1/2Results posted
• A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood PressureCompletedNCT03714776Phase 2Results posted
• Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsCompletedNCT03548415Phase 2Results posted
• Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)CompletedNCT03514420Phase 2Results posted
• Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)CompletedNCT03385239Phase 2Results posted
• Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver DiseaseCompletedNCT03371355Phase 2Results posted
• Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)CompletedNCT03360747Phase 2Results posted
• A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on HemodialysisCompletedNCT03358030Phase 2Results posted
• Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 DiabetesCompletedNCT03334214Phase 2Results posted
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's DiseaseCompletedNCT03186989Phase 1Results posted
• Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular DiseaseCompletedNCT03070782Phase 2Results posted
• The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia SyndromeCompletedNCT02658175Phase 3Results posted
• The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial LipodystrophyTerminatedNCT02527343Phase 2/3Results posted
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's DiseaseCompletedNCT02519036Phase 1/2Results posted
• The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With HypertriglyceridemiaCompletedNCT02300233Phase 3Results posted
• The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia SyndromeCompletedNCT02211209Phase 3Results posted
• Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)CompletedNCT02175004Phase 3Results posted
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)CompletedNCT02160899Phase 2Results posted
• Efficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyCompletedNCT01737398Phase 2/3Results posted
• Zilganersen Expanded Access Program for Individuals With Alexander DiseaseApproved for marketingNCT07487389
• REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeActive, not recruitingNCT06914609Phase 3
• 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)TerminatedNCT06673069Phase 1
• Donidalorsen Expanded Access Program for Patients With Hereditary AngioedemaApproved for marketingNCT06415448
• Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)Approved for marketingNCT06360237
• PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717Active, not recruitingNCT06153966Phase 1/2
• The CARDIO-TTRansform Scintigraphy Sub-studyCompletedNCT06073587
• CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyCompletedNCT06073574
• Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeTerminatedNCT06014541
• CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)Active, not recruitingNCT05681351Phase 3
• A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe HypertriglyceridemiaCompletedNCT05610280Phase 3
• A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult ParticipantsCompletedNCT05579860Phase 1
• A Study of Olezarsen Administered Subcutaneously to Participants With Severe HypertriglyceridemiaCompletedNCT05552326Phase 3
• A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)Active, not recruitingNCT05392114Phase 3
• A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese ParticipantsCompletedNCT05337878Phase 1
• A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled HypertensionCompletedNCT05314439Phase 2
• A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With VolanesorsenActive, not recruitingNCT05185843Phase 3
• A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraCompletedNCT05143957Phase 2
• A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)Active, not recruitingNCT05130450Phase 3
• HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeActive, not recruitingNCT05127226Phase 1/2
• A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe HypertriglyceridemiaCompletedNCT05079919Phase 3
• 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 PolyneuropathyActive, not recruitingNCT05071300Phase 3
• A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547CompletedNCT04934891Phase 1
• A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224CompletedNCT04932512Phase 2
• A Non-interventional Cohort Safety Study of Patients With hATTR-PNActive, not recruitingNCT04850105
• A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)Active, not recruitingNCT04849741Phase 3
• A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection FractionCompletedNCT04836182Phase 2
• 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)Active, not recruitingNCT04768972Phase 3
• A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904CompletedNCT04731623Phase 1
• A Study of ION537 in Patients With Molecularly Selected Advanced Solid TumorsCompletedNCT04659096Phase 1
• A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaCompletedNCT04398485Phase 1
• 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 ParticipantsCompletedNCT04302064Phase 1
• 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)CompletedNCT04136171Phase 3
• 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 NephropathyCompletedNCT04014335Phase 2
• A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseCompletedNCT03976349Phase 1
• Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsCompletedNCT03967249Phase 2
• Natural History and Functional Status Study of Patients With Lafora DiseaseCompletedNCT03876522
• 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)CompletedNCT03815825Phase 2
• 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 FibrosisCompletedNCT03647228Phase 1
• A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy VolunteersCompletedNCT03582462Phase 1
• Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)No longer availableNCT03544060
• Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy PatientsCompletedNCT03506854Phase 1
• Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)WithdrawnNCT03455777Phase 2
• 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)WithdrawnNCT03446144Phase 2
• Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on WarfarinCompletedNCT03426033Phase 1
• ATTR Expanded Access Program (EAP) by IonisApproved for marketingNCT03400098
• Drug-drug Interaction Study to Evaluate the Effect of ISIS 681257 on ClopidogrelCompletedNCT03392051Phase 1
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy VolunteersCompletedNCT03263507Phase 1
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersCompletedNCT03165864Phase 1
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy VolunteersCompletedNCT03101878Phase 1
• A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy VolunteersCompletedNCT02910635Phase 1
• Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated TriglyceridesCompletedNCT02900027Phase 1
• Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 DiabetesCompletedNCT02824003Phase 2
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial HypercholesterolemiaCompletedNCT02709850Phase 1
• Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 DiabetesCompletedNCT02583919Phase 2
• A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on HemodialysisCompletedNCT02553889Phase 2
• A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese PatientsCompletedNCT02476019Phase 2
• Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)CompletedNCT02414594Phase 1

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