Notable
CHAMPION · Obudanersen (ION582) in Children and · Angelman Syndrome (AS)
Recruitment opened
Ionis Pharmaceuticals, Inc.Phase 3 NCT07782827 Registry history of NCT07782827 on ClinicalTrials.gov (opens the registry)
Updated
22 active trials 9 recruiting 12 Phase 3 3 disease areas 5 changes in 30 days
4 trials changed in the last 30 days: 0 stopped, 1 with a notable change, 3 with minor changes only.
Recruitment opened
Ionis Pharmaceuticals, Inc.Phase 3 NCT07782827 Registry history of NCT07782827 on ClinicalTrials.gov (opens the registry)
Updated
Enrollment closed, study ongoing
Ionis Pharmaceuticals, Inc.Phase 3 NCT06914609 Registry history of NCT06914609 on ClinicalTrials.gov (opens the registry)
Updated
Trial completed
Ionis Pharmaceuticals, Inc.Not specified NCT06073574 Registry history of NCT06073574 on ClinicalTrials.gov (opens the registry)
Updated
Primary completion pushed from Feb 2027 to Apr 2027
Also in this update: Completion pushed from Jun 2030 to Aug 2030
Ionis Pharmaceuticals, Inc.Phase 1/2 NCT06153966 Registry history of NCT06153966 on ClinicalTrials.gov (opens the registry)
Updated
| Phase 1 | 2 | |
| Phase 1/2 | 4 | |
| Phase 2 | 1 | |
| Phase 3 | 12 |
| NCT ID | Title | Condition | Status | Updated |
|---|---|---|---|---|
| Phase 3 | ||||
| NCT07727538 | A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in… | Familial Chylomicronemia Syndrome | Recruiting | 2026-10-08 |
| NCT07782827 | CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children… | Angelman Syndrome | Recruiting | 2026-09-28 |
| NCT07298447 | Donidalorsen Treatment in Children With Hereditary Angioedema | Hereditary Angioedema (HAE) | Recruiting | 2026-09-02 |
| NCT06914609 | REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | Angelman Syndrome | Active, Not Recruiting | 2026-09-29 |
| NCT04849741 | A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With… | Alexander Disease | Active, Not Recruiting | 2026-08-21 |
| NCT04768972 | FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics… | Amyotrophic Lateral Sclerosis | Active, Not Recruiting | 2026-07-21 |
| NCT05667493 | An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With… | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Enrolling by Invitation | 2026-04-17 |
| NCT05130450 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial… | Familial Chylomicronemia Syndrome | Active, Not Recruiting | 2025-12-12 |
| NCT05185843 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With… | Familial Chylomicronemia Syndrome | Active, Not Recruiting | 2025-12-12 |
| NCT05392114 | A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic… | Hereditary Angioedema | Active, Not Recruiting | 2025-12-12 |
| NCT05071300 | A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as… | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Active, Not Recruiting | 2025-12-04 |
| NCT05681351 | CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants… | Severe Hypertriglyceridemia | Active, Not Recruiting | 2025-12-01 |
| Phase 2 | ||||
| NCT07604974 | A Study to Assess the Safety, Tolerability and Efficacy of ION775 | Hypertriglyceridemia | Recruiting | 2026-09-03 |
| Phase 1/2 | ||||
| NCT06430385 | ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and… | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Recruiting | 2026-10-08 |
| NCT07531745 | ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome | Dravet Syndrome | Recruiting | 2026-09-03 |
| NCT06153966 | PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and… | Prion Disease | Active, Not Recruiting | 2026-09-18 |
| NCT05127226 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple… | Angelman Syndrome | Active, Not Recruiting | 2026-08-20 |
| Phase 1 | ||||
| NCT04165486 | Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered… | Multiple System Atrophy | Recruiting | 2026-04-27 |
| NCT06150716 | Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of… | Pelizaeus-Merzbacher Disease | Recruiting | 2025-12-12 |
| Not specified | ||||
| NCT05659901 | Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants… | Pelizaeus-Merzbacher Disease | Recruiting | 2026-10-02 |
| NCT04850105 | A Non-interventional Cohort Safety Study of Patients With hATTR-PN | Hereditary Transthyretin Amyloidosis With Polyneuropthy | Active, Not Recruiting | 2026-07-29 |
| NCT04270058 | TEGSEDI Pregnancy Surveillance Program | Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy | Not Yet Recruiting | 2025-08-19 |
Amyloidosis (4) · ALS (1) · Epilepsy (1)