| Phase 3 |
| NCT07727538 |
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in… |
Familial Chylomicronemia Syndrome |
Recruiting |
2026-08-26 |
| NCT06914609 |
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome |
Angelman Syndrome |
Recruiting |
2026-08-12 |
| NCT07298447 |
Donidalorsen Treatment in Children With Hereditary Angioedema |
Hereditary Angioedema (HAE) |
Recruiting |
2026-08-05 |
| NCT07782827 |
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children… |
Angelman Syndrome |
Not Yet Recruiting |
2026-08-24 |
| 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 |
2026-06-18 |
| Phase 1/2 |
| NCT07531745 |
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome |
Dravet Syndrome |
Recruiting |
2026-08-05 |
| NCT06430385 |
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and… |
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome |
Recruiting |
2026-03-24 |
| NCT06153966 |
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and… |
Prion Disease |
Active, Not Recruiting |
2026-08-26 |
| 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 |
2026-04-17 |
| NCT04850105 |
A Non-interventional Cohort Safety Study of Patients With hATTR-PN |
Hereditary Transthyretin Amyloidosis With Polyneuropthy |
Active, Not Recruiting |
2026-07-29 |
| NCT06073574 |
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study |
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) |
Active, Not Recruiting |
2025-11-28 |
| NCT06073587 |
The CARDIO-TTRansform Scintigraphy Sub-study |
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) |
Active, Not Recruiting |
2025-11-28 |
| NCT04270058 |
TEGSEDI Pregnancy Surveillance Program |
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy |
Not Yet Recruiting |
2025-08-19 |