Clinical Trials - IONS

NCT IDTitleStatusPhasesStart DateCompletion DatePrimary Completion Date
NCT06914609REVEAL: A Phase 3 Study of ION582 in Angelman SyndromeRECRUITINGPHASE32025-06-102030-042027-08
NCT06673069Hero: 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)ACTIVE_NOT_RECRUITINGPHASE12024-12-202027-032027-02
NCT06430385ATTUNE: 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)RECRUITINGPHASE1, PHASE22024-10-212030-042027-09
NCT06150716Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)RECRUITINGPHASE12024-04-102028-062028-06
NCT06153966PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717ACTIVE_NOT_RECRUITINGPHASE1, PHASE22024-01-042027-072025-07
NCT05681351CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)RECRUITINGPHASE32022-12-132027-032027-03
NCT05667493An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)ENROLLING_BY_INVITATIONPHASE32022-11-302029-082029-08
NCT05610280A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe HypertriglyceridemiaCOMPLETEDPHASE32022-11-212025-06-092024-08-08
NCT05579860A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult ParticipantsCOMPLETEDPHASE12022-10-052023-05-222023-05-22
NCT05552326A Study of Olezarsen Administered Subcutaneously to Participants With Severe HypertriglyceridemiaCOMPLETEDPHASE32022-08-312025-09-122024-12-17
NCT04165486Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)RECRUITINGPHASE12022-07-212027-092027-09
NCT05392114A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)ACTIVE_NOT_RECRUITINGPHASE32022-07-132027-032026-12
NCT05355402A 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 HypertriglyceridemiaCOMPLETEDPHASE22022-05-092023-12-212023-03-22
NCT05314439A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled HypertensionCOMPLETEDPHASE22022-04-292023-02-152023-01-31
NCT05185843A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With VolanesorsenACTIVE_NOT_RECRUITINGPHASE32022-02-252027-062027-06
NCT05071300A 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_RECRUITINGPHASE32022-01-042029-082029-08
NCT05143957A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraACTIVE_NOT_RECRUITINGPHASE22021-12-302026-062025-06-26
NCT05127226HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeRECRUITINGPHASE1, PHASE22021-12-222029-032029-03
NCT05139810OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)COMPLETEDPHASE32021-12-032023-11-092023-11-09
NCT05130450A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)ACTIVE_NOT_RECRUITINGPHASE32021-11-182028-022028-02
NCT05079919A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe HypertriglyceridemiaCOMPLETEDPHASE32021-10-252025-07-082024-10-22
NCT04932512A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224COMPLETEDPHASE22021-06-172024-02-282024-01-10
NCT04768972FUSION: 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_RECRUITINGPHASE32021-06-142028-032026-06
NCT04836182A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection FractionCOMPLETEDPHASE22021-06-082023-01-112022-10-19
NCT04849741A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)ACTIVE_NOT_RECRUITINGPHASE32021-06-012029-092025-09
NCT04714320A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood PressureCOMPLETEDPHASE22021-04-282022-10-032022-06-29
NCT04934891A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547COMPLETEDPHASE12021-03-242022-09-142022-09-14
NCT04731623A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904COMPLETEDPHASE12021-02-262021-09-112021-08-12
NCT04398485A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaCOMPLETEDPHASE12021-01-202024-09-302024-09-30
NCT04659096A Study of ION537 in Patients With Molecularly Selected Advanced Solid TumorsCOMPLETEDPHASE12021-01-052022-10-192022-10-19
NCT04522180A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyCOMPLETEDPHASE22021-01-042023-05-042022-07-15
NCT04441788A 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)TERMINATEDPHASE22020-12-222021-08-092021-04-27
NCT04568434A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)COMPLETEDPHASE32020-11-182023-10-172023-07-14
NCT04059406Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)TERMINATEDPHASE22020-09-242023-03-282023-03-28
NCT04302064A 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 ParticipantsCOMPLETEDPHASE12020-04-232020-09-102020-09-10
NCT04307381An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary AngioedemaCOMPLETEDPHASE22020-04-012025-01-242025-01-24
NCT04136171CARDIO-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)ACTIVE_NOT_RECRUITINGPHASE32020-03-132026-082026-04
NCT04030598A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary AngioedemaCOMPLETEDPHASE22020-01-072021-03-012021-01-04
NCT04136184NEURO-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 PolyneuropathyCOMPLETEDPHASE32019-12-112023-07-122023-04-11
NCT04014335A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA NephropathyCOMPLETEDPHASE22019-12-042024-04-112024-02-08
NCT04083222A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRxCOMPLETEDPHASE22019-11-132020-07-202020-07-20
NCT03976349A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseCOMPLETEDPHASE12019-08-122024-08-122024-08-12
NCT03967249Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsCOMPLETEDPHASE22019-07-252022-07-072022-03-31
NCT03815825GOLDEN 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)COMPLETEDPHASE22019-03-042024-06-122024-04-18
NCT03714776A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood PressureCOMPLETEDPHASE22019-01-032019-11-132019-11-13
NCT03728634Evaluate 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 ).COMPLETEDPHASE1, PHASE22018-12-212020-02-202020-02-20
NCT03647228A 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 FibrosisCOMPLETEDPHASE12018-12-132020-10-132020-10-13
NCT05337878A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese ParticipantsCOMPLETEDPHASE12018-10-152019-12-182019-12-18
NCT03548415Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsCOMPLETEDPHASE22018-09-132021-04-022021-02-18
NCT03582462A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy VolunteersCOMPLETEDPHASE12018-07-092019-04-262019-04-26
NCT03446144Safety 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)WITHDRAWNPHASE22018-03-162018-10-102018-10-10
NCT03358030A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on HemodialysisCOMPLETEDPHASE22017-12-262019-07-102019-07-10
NCT03334214Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 DiabetesCOMPLETEDPHASE22017-11-032018-11-282018-11-28
NCT03186989Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's DiseaseCOMPLETEDPHASE12017-10-122022-05-122022-05-12
NCT03263507Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy VolunteersCOMPLETEDPHASE12017-08-282018-10-092018-10-09
NCT03165864Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersCOMPLETEDPHASE12017-05-092018-09-252018-09-25
NCT03101878Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy VolunteersCOMPLETEDPHASE12017-04-052018-08-012018-03-29
NCT02910635A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy VolunteersCOMPLETEDPHASE12016-09-192016-12-202016-12-20
NCT02900027Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated TriglyceridesCOMPLETEDPHASE12016-092018-04-302018-04-30
NCT02824003Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 DiabetesCOMPLETEDPHASE22016-042017-05-222016-12-15
NCT02709850Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial HypercholesterolemiaCOMPLETEDPHASE12015-11-302017-06-262017-04-12
NCT02553889A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on HemodialysisCOMPLETEDPHASE22015-102016-112016-11
NCT02583919Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 DiabetesCOMPLETEDPHASE22015-092017-05-112016-12-15
NCT02519036Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's DiseaseCOMPLETEDPHASE1, PHASE22015-08-062017-11-082017-11-08
NCT02476019A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese PatientsCOMPLETEDPHASE22015-062016-092016-07
NCT02414594Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)COMPLETEDPHASE12015-042016-022016-02
NCT02300233The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With HypertriglyceridemiaCOMPLETEDPHASE32015-02-052017-01-242016-07-27
NCT02312011A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1COMPLETEDPHASE1, PHASE22014-122016-082016-07
NCT02211209The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia SyndromeCOMPLETEDPHASE32014-122017-03-282016-12-19
NCT02175004Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)COMPLETEDPHASE32014-06-262021-01-072020-09-11
NCT02160899Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)COMPLETEDPHASE22014-062015-112015-11
NCT01968265Safety, Tolerability and Efficacy of ISIS-GCCRRx in Type 2 DiabetesCOMPLETEDPHASE22013-102015-052015-01
NCT01918865Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 DiabetesCOMPLETEDPHASE22013-082015-022014-10
NCT01885260Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 DiabetesCOMPLETEDPHASE22013-072014-112014-05
NCT01737398Efficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyCOMPLETEDPHASE2, PHASE32013-03-152017-11-072017-03-03
NCT01710852Antiarrhythmic and Symptomatic Effect of ISIS CRP Rx Targeting CRP in Paroxysmal Atrial FibrillationCOMPLETEDPHASE22012-102014-042014-04
NCT01713361Active Comparator-Controlled Study to Assess Safety and Efficacy of ISIS-FXIRx in Total Knee ArthroplastyCOMPLETEDPHASE22012-102014-082014-05
NCT01647308Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 DiabetesTERMINATEDPHASE22012-072014-032014-01
NCT01563302Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced CancersCOMPLETEDPHASE1, PHASE22012-02-272016-03-232016-03-23
NCT01529424Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in HypertriglyceridemiaCOMPLETEDPHASE22012-022014-012014-01
NCT01414101Safety, Tolerability, and Pharmacodynamic Study of ISIS CRP Rx in Rheumatoid ArthritisCOMPLETEDPHASE22011-082013-052013-05
NCT01234025Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC)COMPLETEDPHASE1, PHASE22010-112013-122013-05
NCT01234038Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and PaclitaxelCOMPLETEDPHASE1, PHASE22010-112013-012012-10
NCT01041222Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene MutationsCOMPLETEDPHASE12010-012012-012011-12
NCT00836225Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy VolunteersCOMPLETEDPHASE12009-012012-012011-10
NCT00734240Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy VolunteersCOMPLETEDPHASE12008-072010-032010-02
NCT00519727Safety Study of ISIS 325568 in Healthy VolunteersCOMPLETEDPHASE12007-082008-062008-04
NCT00455598Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With SulfonylureaCOMPLETEDPHASE22007-022009-052009-03
NCT00365781Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715COMPLETEDPHASE12006-082006-09
NCT00327626Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Being Treated With SulfonylureaWITHDRAWNPHASE22006-05-012007-03-012007-02-01
NCT00330200Effects of ISIS 113715 on Insulin Sensitivity, Glucose, and Lipid Metabolism and Energy Expenditure in Type 2 DiabeticsTERMINATEDPHASE22005-11-012007-03-012007-03-01
NCT00062816Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Responding Adequately to the Two DrugsCOMPLETEDPHASE1, PHASE22003-062004-012004-01
NCT00063830ISIS 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.COMPLETEDPHASE22003-04-032004-10-112004-10-11
NCT00330330Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Who Have Not Received Prior TreatmentCOMPLETEDPHASE22003-022006-08
NCT00063414ISIS 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 ColitisCOMPLETEDPHASE22002-11-202005-03-242005-03-24
NCT00048295Alicaforsen (ISIS 2302) in Patients With Active Crohn's DiseaseCOMPLETEDPHASE32002-05
NCT00048321ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid ArthritisCOMPLETEDPHASE22002-01-012003-02-012003-02-01
NCT00035945ISIS 14803-CS2, Treatment With ISIS 14803, Administered IV in Patients With Chronic Hepatitis C Virus InfectionsCOMPLETEDPHASE22001-10-012004-05-012004-05-01
NCT00048113Alicaforsen (ISIS 2302) in Patients With Active Crohn's DiseaseCOMPLETEDPHASE32001-01-102002-04-272002-04-27
NCT00017407Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung CancerCOMPLETEDPHASE32000-10-132002-09-092002-09-09
NCT00002356The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the EyesCOMPLETEDPHASE2
NCT00002156A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )COMPLETEDPHASE2
NCT00002187A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus RetinitisCOMPLETEDNA
NCT00002355A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the EyesCOMPLETEDNA