Our grouping. This page groups the registry's lead sponsor names by company — our grouping, not the registry's. It can be wrong or incomplete: an acquired company whose name never mentions its owner may be missing. Use “Something wrong?” under any name below to correct it.
Tell us about a missing company
Ionis Pharmaceuticals, Inc. clinical trials
The registry lists 133 studies whose lead sponsor is Ionis Pharmaceuticals, Inc. or one of 1 company grouped under it. 11 are recruiting or about to start. Per ClinicalTrials.gov, last updated Oct 20, 2026.
Keep track of what changes. Save the Ionis Pharmaceuticals, Inc. studies recruiting or about to start, and get an email each week — studies that open, close, add sites or post results.
Watch these studiesRegistry sponsor names on this page
Each name exactly as ClinicalTrials.gov lists it, and how it relates to Ionis Pharmaceuticals, Inc..
- Ionis Pharmaceuticals, Inc.118 studiesSame company
Something wrong?
- Akcea Therapeutics15 studies →Subsidiary
Something wrong?
Focus areas
Conditions Ionis Pharmaceuticals, Inc. leads ten or more studies in, by study count. A broader condition is left out when almost all its studies are already in a narrower one listed here.
Studies
Recruiting first, then most recently updated. Showing 50 of 133. Browse all 133 →
- CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)RecruitingNCT07782827Phase 3
- A Study to Assess the Safety, Tolerability and Efficacy of ION775RecruitingNCT07604974Phase 2
- 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
- A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsRecruitingNCT07727538Phase 3
- Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseRecruitingNCT05659901
- ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeRecruitingNCT07531745Phase 1/2
- Donidalorsen Treatment in Children With Hereditary AngioedemaRecruitingNCT07298447Phase 3
- Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)RecruitingNCT04165486Phase 1
- An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)Enrolling by invitationNCT05667493Phase 3
- Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)RecruitingNCT06150716Phase 1
- TEGSEDI Pregnancy Surveillance ProgramNot yet recruitingNCT04270058
Sponsor listed in the registry: Akcea Therapeutics
- REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeActive, not recruitingNCT06914609Phase 3
- Zilganersen Expanded Access Program for Individuals With Alexander DiseaseApproved for marketingNCT07487389
- PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717Active, not recruitingNCT06153966Phase 1/2
- CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyCompletedNCT06073574
- The CARDIO-TTRansform Scintigraphy Sub-studyCompletedNCT06073587
- Donidalorsen Expanded Access Program for Patients With Hereditary AngioedemaApproved for marketingNCT06415448
- 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 Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)Active, not recruitingNCT04849741Phase 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 Non-interventional Cohort Safety Study of Patients With hATTR-PNActive, not recruitingNCT04850105
Sponsor listed in the registry: Akcea Therapeutics
- 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 Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraCompletedNCT05143957Phase 2
- An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary AngioedemaCompletedNCT04307381Phase 2
- 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 2
- Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeTerminatedNCT06014541
- 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
- A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe HypertriglyceridemiaCompletedNCT05610280Phase 3
- A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe HypertriglyceridemiaCompletedNCT05079919Phase 3
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)Active, not recruitingNCT05130450Phase 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 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 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
- CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)Active, not recruitingNCT05681351Phase 3
- A Study of Olezarsen Administered Subcutaneously to Participants With Severe HypertriglyceridemiaCompletedNCT05552326Phase 3
- A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseCompletedNCT03976349Phase 1
- A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PNTerminatedNCT04306510Phase 4
Sponsor listed in the registry: Akcea Therapeutics
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's DiseaseCompletedNCT03186989Phase 1
- 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
- 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 3
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)CompletedNCT04568434Phase 3
- A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood PressureCompletedNCT04714320Phase 2
- Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)TerminatedNCT04059406Phase 2
- 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
- Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)Approved for marketingNCT06360237
- 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 3
- A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224CompletedNCT04932512Phase 2
- A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaCompletedNCT04398485Phase 1
- A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyCompletedNCT04522180Phase 2
- A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult ParticipantsCompletedNCT05579860Phase 1