| A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants (NCT07727538) | Ionis Pharmaceuticals, Inc. | 2026-07-31 |
| ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome (NCT07531745) | Ionis Pharmaceuticals, Inc. | 2026-05-21 |
| A Study to Assess the Safety, Tolerability and Efficacy of ION775 (NCT07604974) | Ionis Pharmaceuticals, Inc. | 2026-05-12 |
| Donidalorsen Treatment in Children With Hereditary Angioedema (NCT07298447) | Ionis Pharmaceuticals, Inc. | 2026-04-21 |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome (NCT06914609) | Ionis Pharmaceuticals, Inc. | 2025-06-10 |
| 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) (NCT06673069) | Ionis Pharmaceuticals, Inc. | 2024-12-20 |
| 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) (NCT06430385) | Ionis Pharmaceuticals, Inc. | 2024-10-21 |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) (NCT06150716) | Ionis Pharmaceuticals, Inc. | 2024-04-10 |
| PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 (NCT06153966) | Ionis Pharmaceuticals, Inc. | 2024-01-04 |
| Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (NCT06014541) | Ionis Pharmaceuticals, Inc. | 2023-10-03 |
| The CARDIO-TTRansform Scintigraphy Sub-study (NCT06073587) | Ionis Pharmaceuticals, Inc. | 2023-04-04 |
| CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG) (NCT05681351) | Ionis Pharmaceuticals, Inc. | 2022-12-13 |
| An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) (NCT05667493) | Ionis Pharmaceuticals, Inc. | 2022-11-30 |
| A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia (NCT05610280) | Ionis Pharmaceuticals, Inc. | 2022-11-21 |
| A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants (NCT05579860) | Ionis Pharmaceuticals, Inc. | 2022-10-05 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease (NCT05659901) | Ionis Pharmaceuticals, Inc. | 2022-10-03 |
| A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (NCT05552326) | Ionis Pharmaceuticals, Inc. | 2022-08-31 |
| Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) (NCT04165486) | Ionis Pharmaceuticals, Inc. | 2022-07-21 |
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) (NCT05392114) | Ionis Pharmaceuticals, Inc. | 2022-07-13 |
| 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 (NCT05355402) | Ionis Pharmaceuticals, Inc. | 2022-05-09 |
| A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension (NCT05314439) | Ionis Pharmaceuticals, Inc. | 2022-04-29 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen (NCT05185843) | Ionis Pharmaceuticals, Inc. | 2022-02-25 |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome (NCT05127226) | Ionis Pharmaceuticals, Inc. | 2021-12-22 |
| OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE) (NCT05139810) | Ionis Pharmaceuticals, Inc. | 2021-12-03 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) (NCT05130450) | Ionis Pharmaceuticals, Inc. | 2021-11-18 |
| A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia (NCT05079919) | Ionis Pharmaceuticals, Inc. | 2021-10-25 |
| A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224 (NCT04932512) | Ionis Pharmaceuticals, Inc. | 2021-06-17 |
| 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) (NCT04768972) | Ionis Pharmaceuticals, Inc. | 2021-06-14 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction (NCT04836182) | Ionis Pharmaceuticals, Inc. | 2021-06-08 |
| A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) (NCT04849741) | Ionis Pharmaceuticals, Inc. | 2021-06-01 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure (NCT04714320) | Ionis Pharmaceuticals, Inc. | 2021-04-28 |
| CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study (NCT06073574) | Ionis Pharmaceuticals, Inc. | 2021-03-31 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547 (NCT04934891) | Ionis Pharmaceuticals, Inc. | 2021-03-24 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904 (NCT04731623) | Ionis Pharmaceuticals, Inc. | 2021-02-26 |
| A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma (NCT04398485) | Ionis Pharmaceuticals, Inc. | 2021-01-20 |
| A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors (NCT04659096) | Ionis Pharmaceuticals, Inc. | 2021-01-05 |
| A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly (NCT04522180) | Ionis Pharmaceuticals, Inc. | 2021-01-04 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (NCT04568434) | Ionis Pharmaceuticals, Inc. | 2020-11-18 |
| Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) (NCT04059406) | Ionis Pharmaceuticals, Inc. | 2020-09-24 |
| 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 (NCT04302064) | Ionis Pharmaceuticals, Inc. | 2020-04-23 |
| An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema (NCT04307381) | Ionis Pharmaceuticals, Inc. | 2020-03-31 |
| 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) (NCT04136171) | Ionis Pharmaceuticals, Inc. | 2020-03-13 |
| A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema (NCT04030598) | Ionis Pharmaceuticals, Inc. | 2020-01-07 |
| 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 (NCT04014335) | Ionis Pharmaceuticals, Inc. | 2019-12-04 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx (NCT04083222) | Ionis Pharmaceuticals, Inc. | 2019-11-13 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease (NCT03976349) | Ionis Pharmaceuticals, Inc. | 2019-08-12 |
| Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands (NCT03967249) | Ionis Pharmaceuticals, Inc. | 2019-07-25 |
| 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) (NCT03815825) | Ionis Pharmaceuticals, Inc. | 2019-03-04 |
| Natural History and Functional Status Study of Patients With Lafora Disease (NCT03876522) | Ionis Pharmaceuticals, Inc. | 2019-01-09 |
| 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 (NCT03714776) | Ionis Pharmaceuticals, Inc. | 2019-01-03 |
| 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 ). (NCT03728634) | Ionis Pharmaceuticals, Inc. | 2018-12-21 |
| 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 (NCT03647228) | Ionis Pharmaceuticals, Inc. | 2018-12-13 |
| A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants (NCT05337878) | Ionis Pharmaceuticals, Inc. | 2018-10-15 |
| Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands (NCT03548415) | Ionis Pharmaceuticals, Inc. | 2018-09-13 |
| 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 (NCT03582462) | Ionis Pharmaceuticals, Inc. | 2018-07-09 |
| 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) (NCT03446144) | Ionis Pharmaceuticals, Inc. | 2018-03-16 |
| A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemodialysis (NCT03358030) | Ionis Pharmaceuticals, Inc. | 2017-12-26 |
| Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes (NCT03334214) | Ionis Pharmaceuticals, Inc. | 2017-11-03 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease (NCT03186989) | Ionis Pharmaceuticals, Inc. | 2017-10-12 |