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Clinical trials
catalyst calendar across sponsors →Studies where IONS is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
108 interventional · 5 observational · 4 expanded access · 23 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeNCT07531745Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEsNon-randomized · Open-label · Treatment | Phase 1/2 | Recruiting | Drug: ION337 | Dravet Syndrome | 32 | Dec 2030 | ≤ 1,595 d | 2026-08-05 |
| A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsNCT07727538Percent Change from Baseline in Fasting Triglycerides (TG)Open-label · Treatment | Phase 3 | Recruiting | Drug: Olezarsen | Familial Chylomicronemia Syndrome | 12 | Aug 2030 | ≤ 1,473 d | 2026-08-18 |
| 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 PolyneuropathyNCT05071300Change From Baseline in Platelet CountOpen-label · Treatment | Phase 3 | Active, not recruiting | Drug: Eplontersen | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | 151 | Aug 2029 | ≤ 1,108 d | 2025-12-04 |
| An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)NCT05667493Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)Open-label · Treatment | Phase 3 | Enrolling by invitation | Drug: Eplontersen | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | 1,400 | Aug 2029 | ≤ 1,108 d | 2026-04-17 |
| Donidalorsen Treatment in Children With Hereditary AngioedemaNCT07298447Number of Participants with Treatment Emergent Adverse Events (TEAEs)Non-randomized · Open-label · Treatment | Phase 3 | Recruiting | Drug: Donidalorsen | Hereditary Angioedema (HAE) | 20 | Jun 2029 | ≤ 1,046 d | 2026-08-05 |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeNCT05127226To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters).Non-randomized · Open-label · Treatment | Phase 1/2 | Recruiting | Drug: ION582 | Angelman Syndrome | 70 | Mar 2029 | ≤ 955 d | 2025-11-14 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseNCT05659901observationalAssess Longitudinal Changes in Fluid Biomarkers | — | Recruiting | — | Pelizaeus-Merzbacher Disease | 32 | Mar 2029 | ≤ 955 d | 2026-04-17 |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)NCT06150716Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEsRandomized · Open-label · Treatment | Phase 1 | Recruiting | Drug: ION356 | Pelizaeus-Merzbacher Disease | 24 | Jun 2028 | ≤ 681 d | 2025-12-12 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)NCT05130450Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to BaselineOpen-label · Treatment | Phase 3 | Active, not recruiting | Drug: Olezarsen | Familial Chylomicronemia Syndrome | 60 | Feb 2028 | ≤ 559 d | 2025-12-12 |
| Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)NCT04165486Number of Participants with Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 1 | Recruiting | Drug: ION464, Placebo | Multiple System Atrophy | 40 | Sep 2027 | ≤ 407 d | 2026-04-27 |
| 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)NCT06430385Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)Randomized · Quadruple-masked · Treatment | Phase 1/2 | Recruiting | Drug: ION440 · Procedure: Sham procedure | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | 48 | Sep 2027 | ≤ 407 d | 2026-03-24 |
| A Study to Assess the Safety, Tolerability and Efficacy of ION775NCT07604974Percent Change From Baseline in Fasting Triglyceride (TG)Randomized · Open-label · Treatment | Phase 2 | Recruiting | Drug: ION775 | Hypertriglyceridemia, Severe Hypertriglyceridemia | 72 | Sep 2027 | ≤ 407 d | 2026-06-18 |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeNCT06914609Change in Performance on the Expressive Communication Subdomain Raw Score of the Bayley Scales for Infant and Toddler Development-4 (Bayley-4) Without Caregiver Input in Cohort 1Randomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: obudanersen, Placebo | Angelman Syndrome | 158 | Aug 2027 | ≤ 377 d | 2026-08-12 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With VolanesorsenNCT05185843Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: Olezarsen | Familial Chylomicronemia Syndrome | 24 | Jun 2027 | ≤ 315 d | 2025-12-12 |
| CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)NCT05681351Proportion of Participants With Change in Clinical Laboratory Values From Baseline to Week 53, From Baseline to Week 105, and From Baseline to Week 157Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: Olezarsen | Severe Hypertriglyceridemia | 885 | Mar 2027 | ≤ 224 d | 2025-12-01 |
| PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717NCT06153966Incidence of treatment-emergent adverse events.Randomized · Double-masked · Treatment | Phase 1/2 | Active, not recruiting | Drug: ION717, Placebo | Prion Disease | 85 | Feb 2027 | ≤ 193 d | 2026-08-19 |
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)NCT05392114Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by SeverityOpen-label · Treatment | Phase 3 | Active, not recruiting | Drug: Donidalorsen | Hereditary Angioedema | 154 | Dec 2026 | ≤ 134 d | 2025-12-12 |
| 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)NCT04768972Change from Baseline (Day 1) through Study Day 505 in Part 1 in functional impairmentRandomized · Double-masked · Treatment | Phase 3 | Active, not recruiting | Drug: ION363, Placebo | Amyotrophic Lateral Sclerosis | 89 | 2026-06-17actual | — | 2026-07-21 |
| 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)NCT04136171Composite Outcome of Cardiovascular (CV) Mortality and Recurrent CV Clinical Events up to Week 140Randomized · Double-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Eplontersen, Placebo | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | 1,438 | Apr 2026 | — | 2025-08-08 |
| CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyNCT06073574observationalChange in the Percent of ECV in Participants Receiving ION-682884 vs. Placebo According to ION-682884-CS2 Treatment Groups | — | Active, not recruiting | Diagnostic test: Cardiac MRI | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | 150 | Mar 2026 | — | 2025-11-28 |
| The CARDIO-TTRansform Scintigraphy Sub-studyNCT06073587observationalChanges From Baseline in Perugini Grading Score From Scintigraphy Scan Images at Week 140 | — | Active, not recruiting | Diagnostic test: Scintigraphy scan | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | 150 | Mar 2026 | — | 2025-11-28 |
| A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)NCT04849741Percent Change from Baseline in the 10-Meter Walk Test (10MWT)Randomized · Double-masked · Treatment | Phase 3 | Active, not recruiting | Drug: zilganersen, Placebo | Alexander Disease | 54 | 2025-08-22actual | — | 2026-04-03 |
| 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)NCT06673069Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Sponsor decision to terminate study early. Decision not based on safety concerns. | Drug: ION269 | Alzheimer Disease, Down Syndrome | 1 | 2025-12-03actual | — | 2026-02-20 |
| Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeNCT06014541observationalChange From Baseline in MeCP2 in the CSF | — | Terminatedsponsor's stated reason: Sponsor decision to terminate study early due to sufficient data collected, the study objectives met and defined unmet need and disease trajectory in males with MECP2 duplication syndrome. | — | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | 29 | 2025-10-07actual | — | 2026-04-09 |
| A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraNCT05143957Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment PeriodRandomized · Open-label · Treatment | Phase 2 | Completed | Drug: sapablursen | Phlebotomy Dependent Polycythemia Vera | 50 | 2025-06-26actual | — | 2026-07-09 |
| An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary AngioedemaNCT04307381results2026-05-08Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Donidalorsen | Hereditary Angioedema | 20 | 2025-01-24actual | — | 2026-05-08 |
| A Study of Olezarsen Administered Subcutaneously to Participants With Severe HypertriglyceridemiaNCT05552326Percent Change from Baseline in Fasting TG Compared to PlaceboRandomized · Triple-masked · Treatment | Phase 3 | Completed | Drug: Olezarsen, Placebo | Severe Hypertriglyceridemia | 446 | 2024-12-17actual | — | 2025-10-22 |
| A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe HypertriglyceridemiaNCT05079919Percent Change from Baseline in Fasting TG Compared to PlaceboRandomized · Triple-masked · Treatment | Phase 3 | Completed | Drug: Olezarsen, Placebo | Severe Hypertriglyceridemia | 617 | 2024-10-22actual | — | 2026-02-10 |
| A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaNCT04398485Maximum-Tolerated Dose (MTD)Open-label · Treatment | Phase 1 | Completed | Drug: ION251 | Relapsed Multiple Myeloma, Refractory Multiple Myeloma | 23 | 2024-09-30actual | — | 2024-10-31 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseNCT03976349Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: BIIB094, Placebo | Parkinson's Disease | 62 | 2024-08-12actual | — | 2025-09-23 |
| A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe HypertriglyceridemiaNCT05610280Percent Change From Baseline to Week 25 in Fasting Triglycerides (TG) Compared to PlaceboRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Olezarsen, Placebo | Hypertriglyceridemia, Cardiovascular Diseases, Atherosclerosis | 1,478 | 2024-08-08actual | — | 2026-02-10 |
| 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)NCT03815825Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal ImagingRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: IONIS-FB-LRx, Placebo | Macular Degeneration, Geographic Atrophy | 332 | 2024-04-18actual | — | 2025-03-07 |
| 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 NephropathyNCT04014335Percent Reduction in 24-hour Urine Protein ExcretionOpen-label · Treatment | Phase 2 | Completed | Drug: IONIS-FB-LRx | Primary IgA Nephropathy | 23 | 2024-02-08actual | — | 2025-01-23 |
| A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224NCT04932512Percentage of Participants With at Least 2-point Reduction in Non-alcoholic Fatty Liver Disease Activity Score (NAS) With at least 1-point Improvement in Hepatocellular Ballooning or Lobular Inflammation, and Without Worsening in Fibrosis Stage at EOTRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ION224 · Other: Placebo | Steatohepatitis, Nonalcoholic | 160 | 2024-01-10actual | — | 2024-11-27 |
| OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)NCT05139810results2025-03-06Time-Normalized Investigator-Confirmed (IC) HAE Attack Rate (Per Month) From Week 1 to Week 25Randomized · Quadruple-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Donidalorsen, Placebo | Hereditary Angioedema | 91 | 2023-11-09actual | — | 2025-03-06 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)NCT04568434results2025-03-06Percent Change From Baseline in Fasting TG at Month 6Randomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Olezarsen, Placebo | Familial Chylomicronemia Syndrome | 66 | 2023-07-14actual | — | 2025-03-06 |
| A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult ParticipantsNCT05579860Area under the plasma concentration-time curve (AUC) of olezarsen from time 0 to 336 hours (AUC0-336h)Randomized · Open-label · Other | Phase 1 | Completed | Drug: Olezarsen | Healthy Participants | 104 | 2023-05-22actual | — | 2024-05-10 |
| 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 PolyneuropathyNCT04136184results2024-12-10Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66Randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Inotersen, Eplontersen | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | 168 | 2023-04-11actual | — | 2024-12-13 |
| Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)NCT04059406results2025-02-18Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27Randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Favorable safety and tolerability were seen, but efficacy results in the mid-stage study did not meet Ionis' minimum target product profile to justify further development. | Drug: sapablursen | Beta Thalassemia Intermedia | 29 | 2023-03-28actual | — | 2025-02-18 |
| 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 HypertriglyceridemiaNCT05355402results2026-04-23Percent Change From Baseline in Fasting TG at Month 6Randomized · Quadruple-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Olezarsen, Placebo | Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease, Severe Hypertriglyceridemia | 154 | 2023-03-22actual | — | 2026-04-23 |
| A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled HypertensionNCT05314439Percent Change from Baseline in Plasma Angiotensinogen (AGT)Randomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: ION904, Placebo | Hypertension | 48 | 2023-01-31actual | — | 2023-12-22 |
| A Study of ION537 in Patients With Molecularly Selected Advanced Solid TumorsNCT04659096Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) and Treatment-emergent Serious Adverse Event (TESAE), Graded by SeverityOpen-label · Treatment | Phase 1 | Completed | Drug: ION537 | Advanced Solid Tumors | 15 | 2022-10-19actual | — | 2022-12-21 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection FractionNCT04836182Percent Change in Plasma AGT Concentration From Baseline to Study Day 85Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: IONIS-AGT-LRx, Placebo | Chronic Heart Failure With Reduced Ejection Fraction | 72 | 2022-10-19actual | — | 2023-09-11 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547NCT04934891Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)Randomized · Double-masked · Treatment | Phase 1 | Completed | Drug: ION547, Placebo | Healthy Volunteers | 48 | 2022-09-14actual | — | 2022-09-23 |
| A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyNCT04522180results2024-10-03Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27Randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: GHR-LRX | Acromegaly | 34 | 2022-07-15actual | — | 2024-10-03 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood PressureNCT04714320results2025-02-18Change From Baseline in Seated Automated Office SBP to Day 85Randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Placebo, IONIS-AGT-LRx | Hypertension | 160 | 2022-06-29actual | — | 2025-02-18 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's DiseaseNCT03186989results2025-04-08Part 1: Number of Participants With Adverse Events That Are Related to Treatment With ISIS 814907Randomized · Double-masked · OtherStudy Protocol and Statistical Analysis Plan ↗ | Phase 1 | Completed | Drug: IONIS MAPTRx · Other: Placebo | Mild Alzheimer's Disease | 46 | 2022-05-12actual | — | 2025-04-08 |
| Natural History and Functional Status Study of Patients With Lafora DiseaseNCT03876522observationalChanges over time in symptom-directed physical exams, measured by height assessment | — | Completed | — | Lafora Disease | 33 | 2022-04-01actual | — | 2022-09-28 |
| Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsNCT03967249The Incidence of Adverse EventsOpen-label · Treatment | Phase 2 | Completed | Drug: IONIS GHR-LRx, Somatostatin Receptor Ligand (SRL) | Acromegaly | 39 | 2022-03-31actual | — | 2023-03-22 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904NCT04731623Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)Randomized · Double-masked · Treatment | Phase 1 | Completed | Drug: ION904, Placebo | Healthy Volunteers | 40 | 2021-08-12actual | — | 2022-08-09 |
| 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)NCT04441788results2022-12-20Change From Baseline to the Primary Time Point in Forced Expiratory Volume in 1 Second (FEV1) Compared to PlaceboRandomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Business Decision to Terminate Study | Drug: ION-827359, Placebo, ION-827359 | Chronic Bronchitis, Chronic Obstructive Pulmonary Disease | 60 | 2021-04-27actual | — | 2022-12-20 |
| Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor LigandsNCT03548415results2022-11-14Percent Change in Serum Insulin-like Growth Factor-1 (IGF-1) From Baseline to 28 Days After Last DoseRandomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: IONIS-GHR-LRx, Placebo | Acromegaly | 43 | 2021-02-18actual | — | 2022-11-14 |
| A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary AngioedemaNCT04030598results2022-09-28Time-normalized Number of HAE Attacks (Per Month) From Week 1 to Week 17Randomized · Triple-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Donidalorsen, Placebo | Hereditary Angioedema | 23 | 2021-01-04actual | — | 2023-04-03 |
| 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 FibrosisNCT03647228Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse EventRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: IONIS-ENaCRx, Placebo | Healthy Subjects, Cystic Fibrosis | 98 | 2020-10-13actual | — | 2021-02-04 |
| Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)NCT02175004results2023-02-09Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study DrugNon-randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Inotersen | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | 135 | 2020-09-11actual | — | 2023-11-18 |
| 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 ParticipantsNCT04302064Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by SeverityRandomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: Eplontersen, Placebo | Healthy Participants | 24 | 2020-09-10actual | — | 2022-03-31 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRxNCT04083222results2023-01-18Percent Change From Baseline in Plasma Angiotensinogen (AGT) at Day 57 Compared to PlaceboRandomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Placebo, ISIS 757456 | Hypertension | 26 | 2020-07-20actual | — | 2023-01-18 |
| 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 ).NCT03728634results2022-12-19Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)Randomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: ION-682884, Placebo · Dietary supplement: Vitamin A | Healthy Volunteers, hATTR Amyloidosis | 47 | 2020-02-20actual | — | 2022-12-19 |
| A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese ParticipantsNCT05337878SAD: Percentage of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Evaluation ParametersRandomized · Quadruple-masked · Other | Phase 1 | Completed | Drug: Placebo, Pelacarsen | Healthy Participants | 29 | 2019-12-18actual | — | 2022-04-20 |
| A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood PressureNCT03714776results2023-01-06Percent Change From Baseline in Plasma Angiotensinogen (AGT) at Day 43 (Week 7) Compared to PlaceboRandomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Placebo, ISIS 757456 | Mild Hypertension | 25 | 2019-11-13actual | — | 2023-01-06 |
| A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on HemodialysisNCT03358030results2023-01-20Number of Participants With Major Bleeding (MB) and Clinically Relevant Non-Major Bleeding (CRNMB)Randomized · Quadruple-masked · PreventionStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: ISIS 416858, Placebo | End-stage Renal Disease (ESRD) | 213 | 2019-07-10actual | — | 2023-01-20 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy VolunteersNCT03582462Safety and Tolerability as Measured by the Number of Participants with Treatment-Emergent Adverse EventsRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: IONIS FXI-LRx, Placebo | Healthy Participants | 66 | 2019-04-26actual | — | 2022-01-18 |
| Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 DiabetesNCT03334214results2020-01-27Absolute Change in Liver Fat Percentage (Randomized Population)Randomized · Double-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: IONIS DGAT2Rx, Placebo | Hepatic Steatosis | 44 | 2018-11-28actual | — | 2020-01-27 |
| 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)NCT03446144Efficacy of IONIS-FB-LrxRandomized · Triple-masked · Treatment | Phase 2 | Withdrawnsponsor's stated reason: Business objective change, no safety or efficacy concerns | Drug: IONIS-FB-Lrx, Placebo (sterline saline 0.9%) | Geographic Atrophy, Age Related Macular Degeneration | 0 | 2018-10-10actual | — | 2019-08-13 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy VolunteersNCT03263507Incidence and severity of adverse events that are related to treatment with DonidalorsenRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: Placebo, Donidalorsen | Healthy Volunteers | 32 | 2018-10-09actual | — | 2022-08-04 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersNCT03165864Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-LrxRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: IONIS TMPRSS6-Lrx · Other: Placebo | Thalassemia | 36 | 2018-09-25actual | — | 2019-01-22 |
| Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated TriglyceridesNCT02900027To evaluate the safety and tolerability of single and multiple doses of IONIS- APOC-III-LRx - (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)Randomized · Double-masked · Other | Phase 1 | Completed | Drug: APOC-III-L-Rx, Placebo Comparator | Elevated Triglycerides (TG) | 56 | 2018-04-30actual | — | 2018-05-22 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy VolunteersNCT03101878Incidence and severity of adverse events that are related to treatment with IONIS AGT-LRxRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: Ionis AGT-LRx, Placebo | Healthy Volunteers | 62 | 2018-03-29actual | — | 2018-08-21 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's DiseaseNCT02519036results2019-05-31Number of Participants With Treatment-related Adverse Events (TEAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: ISIS 443139 10 mg, ISIS 443139 30 mg, ISIS 443139 60 mg, ISIS 443139 90 mg, ISIS 443139 120 mg · Other: Placebo | Huntington's Disease | 46 | 2017-11-08actual | — | 2019-05-31 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial HypercholesterolemiaNCT02709850Safety and tolerability of single and multiple doses of IONIS ANGPTL3-LRx (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)Randomized · Double-masked · Other | Phase 1 | Completed | Drug: IONIS ANGPTL3-LRx, Placebo | Hypertriglyceridemia, Familial Hypercholesterolemia | 48 | 2017-04-12actual | — | 2020-11-19 |
| Efficacy and Safety of Inotersen in Familial Amyloid PolyneuropathyNCT01737398results2019-01-23Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Completed | Drug: Inotersen, Placebo | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | 173 | 2017-03-03actual | — | 2019-07-17 |
| A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy VolunteersNCT02910635Placebo corrected change from baseline in QTcF (corrected Frederica's CT Interval)Randomized · Quadruple-masked · Other | Phase 1 | Completed | Drug: Volanesorsen, Moxifloxacin, Placebo | Abnormalities, Cardiovascular | 52 | 2016-12-20actual | — | 2022-01-28 |
| The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia SyndromeNCT02211209results2022-04-13Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Volanesorsen, Placebo | Familial Chylomicronemia Syndrome | 67 | 2016-12-19actual | — | 2022-04-13 |
| Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 DiabetesNCT02583919Change in HbA1CRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ISIS-GCGRRx- Dose Level 1, ISIS-GCGRRx- Dose Level 2, Placebo | Type 2 Diabetes | 79 | 2016-12-15actual | — | 2018-06-25 |
| Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 DiabetesNCT02824003Change in fasting hepatic glycogen contentRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ISIS-GCGRRx, Placebo | Type 2 Diabetes | 15 | 2016-12-15actual | — | 2018-05-08 |
| A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on HemodialysisNCT02553889Safety and Tolerability - evaluated by reviewing frequency and severity of Adverse events (including bleeding events) and use of concomitant medications, changes in vital signs and laboratory evaluations for all patientsRandomized · Quadruple-masked · Prevention | Phase 2 | Completed | Drug: ISIS 416858, Placebo | End-stage Renal Disease (ESRD) | 49 | Nov 2016actual | — | 2016-12-13 |
| A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1NCT02312011Safety (The number of participants with adverse events)Randomized · Triple-masked · Treatment | Phase 1/2 | Completed | Drug: IONIS-DMPKRx, Placebo | Myotonic Dystrophy Type 1 | 48 | Jul 2016actual | — | 2022-12-06 |
| A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese PatientsNCT02476019Energy Expenditure using both room calorimetry and hood (metabolic cart) calorimetryRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ISIS-FGFR4RX, Placebo | Obesity | 13 | Jul 2016actual | — | 2018-06-25 |
| The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With HypertriglyceridemiaNCT02300233results2022-04-13Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Volanesorsen, Placebo | Hypertriglyceridemia | 114 | 2016-07-27actual | — | 2022-04-13 |
| Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced CancersNCT01563302Safety of IONIS-STAT3Rx in patients with Advanced CancersOpen-label · Treatment | Phase 1/2 | Completed | Drug: IONIS-STAT3Rx | Advanced Cancers, DLBCL, Lymphoma | 64 | 2016-03-23actual | — | 2018-06-25 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)NCT02414594To evaluate the safety and tolerability of single and multiple doses of IONIS-APO(a)-LRx (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)Randomized · Double-masked · Other | Phase 1 | Completed | Drug: IONIS-APO(a)-LRx, Sterile Normal Saline (0.9% NaCl) | Elevated Lipoprotein(a) | 60 | Feb 2016actual | — | 2018-12-05 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)NCT02160899results2019-12-20Percent Change From Baseline in Lipoprotein Lp(a) Plasma Concentration at Day 85/Day 99Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ISIS-APO(a)Rx, Placebo | Elevated Lipoprotein(a) | 64 | Nov 2015actual | — | 2019-12-20 |
| Safety, Tolerability and Efficacy of ISIS-GCCRRx in Type 2 DiabetesNCT01968265The effect of ISIS-GCCRRx on serum fructosamineRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ISIS-GCCRRx, Placebo | Type 2 Diabetes Mellitus | 38 | Jan 2015actual | — | 2015-05-13 |
| Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 DiabetesNCT01918865Incidence, severity, dose-relationship of adverse effects, and changes in laboratory evaluations as a measure of safetyRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: ISIS-PTP1BRx, Placebo, daily OAD (metformin and/or sulfonylurea) | Type 2 Diabetes Mellitus, Obese | 92 | Oct 2014actual | — | 2015-03-03 |
| Active Comparator-Controlled Study to Assess Safety and Efficacy of ISIS-FXIRx in Total Knee ArthroplastyNCT01713361Primary efficacy outcomeRandomized · Open-label · Prevention | Phase 2 | Completed | Drug: ISIS-FXIRx Dose #2, ISIS-FXIRx Dose #3, Enoxaparin | Venous Thromboembolism | 315 | May 2014actual | — | 2014-08-27 |
| Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 DiabetesNCT01885260The effect of ISIS-GCGRRx on serum fructosamineRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: ISIS-GCGRRx - Dose Level 1, ISIS-GCGRRx - Dose Level 2, Placebo | Type 2 Diabetes Mellitus | 77 | May 2014actual | — | 2016-07-04 |
| Antiarrhythmic and Symptomatic Effect of ISIS CRP Rx Targeting CRP in Paroxysmal Atrial FibrillationNCT01710852AF burden reductionRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: ISIS CRP Rx, Placebo | Paroxysmal Atrial Fibrillation | 7 | Apr 2014actual | — | 2015-08-26 |
| Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in HypertriglyceridemiaNCT01529424VLDL apoC-IIIRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ISIS apoC-III Rx, ISIS apoC-III Rx, ISIS apoC-III Rx, Placebo, Placebo, Placebo | Hypertriglyceridemia | 89 | Jan 2014actual | — | 2022-01-27 |
| Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 DiabetesNCT01647308Total apoC-IIIRandomized · Quadruple-masked · Treatment | Phase 2 | Terminatedsponsor's stated reason: The study was terminated for business planning purposes. Assessments of APOCIIIRx in this population will be explored in a larger efficacy study | Drug: ISIS-APOCIIIRX, Placebo | Type 2 Diabetes | 15 | Jan 2014actual | — | 2022-01-18 |
| Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC)NCT01234025Progression free survivalRandomized · Open-label · Treatment | Phase 1/2 | Completed | Drug: ISIS EIF4E Rx, ISIS EIF4E Rx, ISIS EIF4E Rx, Prednisone, Docetaxel | Castrate-Resistant Prostate Cancer | 113 | May 2013actual | — | 2023-10-06 |
| Safety, Tolerability, and Pharmacodynamic Study of ISIS CRP Rx in Rheumatoid ArthritisNCT01414101SafetyRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: ISIS CRP Rx or Placebo | Rheumatoid Arthritis | 51 | May 2013actual | — | 2013-05-17 |
| Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and PaclitaxelNCT01234038Progression free survivalRandomized · Open-label · Treatment | Phase 1/2 | Completed | Drug: ISIS EIF4E Rx, ISIS EIF4E Rx, ISIS EIF4E Rx, Paclitaxel, Carboplatin | Non-small Cell Lung Cancer | 116 | Oct 2012actual | — | 2018-06-27 |
| Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene MutationsNCT01041222To evaluate the safety, tolerability, and pharmacokinetics of four dose levels of ISIS 333611Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: ISIS 333611 | Familial Amyotrophic Lateral Sclerosis | 33 | Dec 2011actual | — | 2012-04-13 |
| Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy VolunteersNCT00836225To evaluate the safety and tolerability of single and multiple doses of ISIS-SGLT2Rx administered subcutaneouslyRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: ISIS 388626 | Type 2 Diabetes Mellitus | 103 | Oct 2011actual | — | 2012-02-03 |
| Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy VolunteersNCT00734240To evaluate the safety and tolerability of single and multiple doses of ISIS 353512 administered intravenously and subcutaneously.Randomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: 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 | Inflammatory Diseases | 103 | Feb 2010actual | — | 2010-07-29 |
| Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With SulfonylureaNCT00455598Change and % change from baseline HbA1cRandomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: ISIS 113715 | Type 2 Diabetes Mellitus | 88 | Mar 2009actual | — | 2009-08-28 |
| Safety Study of ISIS 325568 in Healthy VolunteersNCT00519727safety and tolerabilityRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: ISIS 325568 | Type 2 Diabetes Mellitus | 60 | Apr 2008actual | — | 2008-08-19 |
| Effects of ISIS 113715 on Insulin Sensitivity, Glucose, and Lipid Metabolism and Energy Expenditure in Type 2 DiabeticsNCT00330200Evaluate the effects of ISIS 113715 on hepatic and peripheral insulin sensitivityRandomized · Quadruple-masked · Treatment | Phase 2 | Terminatedsponsor's stated reason: Due to poor enrollment | Drug: ISIS 113715 | Type 2 Diabetes Mellitus | 2 | 2007-03-01actual | — | 2022-12-05 |
| Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Being Treated With SulfonylureaNCT00327626Evaluate the safety, tolerability, and pharmacokinetics of two ISIS 113715 subcutaneous dosages in combination with OAD versus OAD + placebo.Randomized · Double-masked · Treatment | Phase 2 | Withdrawnsponsor's stated reason: study was never started due to regional geopolitical conflict | Drug: ISIS 113715 | Type 2 Diabetes Mellitus | 0 | 2007-02-01actual | — | 2022-12-05 |
| 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 ColitisNCT00063414Treatment | Phase 2 | Completed | Drug: Alicaforsen | Ulcerative Colitis | — | 2005-03-24actual | — | 2022-12-05 |
| 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.NCT00063830Treatment | Phase 2 | Completed | Drug: Alicaforsen | Ulcerative Colitis | — | 2004-10-11actual | — | 2022-12-05 |
| ISIS 14803-CS2, Treatment With ISIS 14803, Administered IV in Patients With Chronic Hepatitis C Virus InfectionsNCT00035945Treatment | Phase 2 | Completed | Drug: ISIS 14803 | Hepatitis C, Chronic | — | 2004-05-01actual | — | 2022-12-05 |
| Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Responding Adequately to the Two DrugsNCT00062816Treatment | Phase 1/2 | Completed | Drug: ISIS 14803, peginterferon alfa, ribavirin | Hepatitis C, Chronic | 22 | Jan 2004actual | — | 2022-12-05 |
| ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid ArthritisNCT00048321Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ISIS 104838 | Rheumatoid Arthritis | 160 | 2003-02-01actual | — | 2022-12-05 |
| Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung CancerNCT00017407Treatment | Phase 3 | Completed | Biological: ISIS 3521 · Drug: carboplatin, paclitaxel | Lung Cancer | — | 2002-09-09actual | — | 2022-12-05 |
| Alicaforsen (ISIS 2302) in Patients With Active Crohn's DiseaseNCT00048113Randomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Alicaforsen | Crohn's Disease | 150 | 2002-04-27actual | — | 2022-12-05 |
| A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )NCT00002156Open-label · Treatment | Phase 2 | Completed | Drug: Fomivirsen sodium, Ganciclovir | Cytomegalovirus Retinitis, HIV Infections | 194 | — | — | 2005-06-24 |
| A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus RetinitisNCT00002187Treatment | Not applicable | Completed | Drug: Fomivirsen sodium | Cytomegalovirus Retinitis, HIV Infections | — | — | — | 2005-06-24 |
| A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the EyesNCT00002355Treatment | Not applicable | Completed | Drug: Fomivirsen sodium | Cytomegalovirus Retinitis, HIV Infections | 60 | — | — | 2005-06-24 |
| The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the EyesNCT00002356Open-label · Treatment | Phase 2 | Completed | Drug: Fomivirsen sodium | Cytomegalovirus Retinitis, HIV Infections | — | — | — | 2005-06-24 |
| Alicaforsen (ISIS 2302) in Patients With Active Crohn's DiseaseNCT00048295Randomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Alicaforsen | Crohn's Disease | 150 | — | — | 2022-08-15 |
| Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Who Have Not Received Prior TreatmentNCT00330330To evaluate the safety and tolerability of ISIS 113715Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: ISIS 113715 | Type 2 Diabetes Mellitus | 96 | — | — | 2008-02-06 |
| Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715NCT00365781Compare the dermatologic responses to 200 mg/1.0 mL and 200 mg/2.0 mL ISIS 113715 administered subcutaneously into separate abdominal sites, with both injections given on the same day.Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: ISIS 113715 | Type 2 Diabetes Mellitus | 10 | — | — | 2007-10-17 |
| ATTR Expanded Access Program (EAP) by IonisNCT03400098expanded access | — | Approved for marketing | Drug: Inotersen | Amyloidosis, Hereditary | — | — | — | 2019-08-05 |
| Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)NCT06360237expanded access | — | Approved for marketing | Drug: Olezarsen | Familial Chylomicronemia Syndrome | — | — | — | 2024-12-27 |
| Donidalorsen Expanded Access Program for Patients With Hereditary AngioedemaNCT06415448expanded access | — | Available | Drug: Donidalorsen | Hereditary Angioedema | — | — | — | 2026-08-05 |
| Zilganersen Expanded Access Program for Individuals With Alexander DiseaseNCT07487389expanded access | — | Available | Drug: zilganersen | Alexander Disease | — | — | — | 2026-03-23 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19