| Detected | Trial | Change |
|---|---|---|
| 2026-08-06 | NCT07298447 Donidalorsen Treatment in Children With Hereditary Angioedema |
Trial sites expanded: 10 -> 13 locations |
| 2026-07-30 | NCT04850105 A Non-interventional Cohort Safety Study of Patients With hATTR-PN |
Enrollment closed, study ongoing |
| 2026-07-30 | NCT04850105 A Non-interventional Cohort Safety Study of Patients With hATTR-PN |
Enrollment reduced: 240 -> 202 participants |
| First Posted | Trial | Phase | Status |
|---|---|---|---|
| 2026-07-27 | NCT07727538 A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in… | Phase 3 | Recruiting |
| NCT ID | Title | Condition | Status |
|---|---|---|---|
| Phase 3 | |||
| NCT07727538 | A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in… | Familial Chylomicronemia Syndrome | Recruiting |
| NCT07298447 | Donidalorsen Treatment in Children With Hereditary Angioedema | Hereditary Angioedema (HAE) | Recruiting |
| NCT06914609 | REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | Angelman Syndrome | Recruiting |
| NCT04136171 | CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly… | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Active, Not Recruiting |
| NCT05681351 | CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants… | Severe Hypertriglyceridemia | Active, Not Recruiting |
| NCT05071300 | A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as… | Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Active, Not Recruiting |
| NCT05130450 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial… | Familial Chylomicronemia Syndrome | Active, Not Recruiting |
| NCT05185843 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With… | Familial Chylomicronemia Syndrome | Active, Not Recruiting |
| NCT05392114 | A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic… | Hereditary Angioedema | Active, Not Recruiting |
| NCT04849741 | A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With… | Alexander Disease | Active, Not Recruiting |
| NCT05667493 | An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With… | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Enrolling by Invitation |
| NCT04768972 | FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics… | Amyotrophic Lateral Sclerosis | Active, Not Recruiting |
| Phase 2 | |||
| NCT07604974 | A Study to Assess the Safety, Tolerability and Efficacy of ION775 | Hypertriglyceridemia | Recruiting |
| Phase 1/2 | |||
| NCT05127226 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple… | Angelman Syndrome | Recruiting |
| NCT06430385 | ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and… | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Recruiting |
| NCT06153966 | PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and… | Prion Disease | Recruiting |
| NCT07531745 | ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome | Dravet Syndrome | Recruiting |
| Phase 1 | |||
| NCT06150716 | Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of… | Pelizaeus-Merzbacher Disease | Recruiting |
| NCT04165486 | Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered… | Multiple System Atrophy | Recruiting |
| Not specified | |||
| NCT05659901 | Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants… | Pelizaeus-Merzbacher Disease | Recruiting |
| NCT04270058 | TEGSEDI Pregnancy Surveillance Program | Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy | Not Yet Recruiting |
| NCT06073574 | CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Active, Not Recruiting |
| NCT06073587 | The CARDIO-TTRansform Scintigraphy Sub-study | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | Active, Not Recruiting |
| NCT04850105 | A Non-interventional Cohort Safety Study of Patients With hATTR-PN | Hereditary Transthyretin Amyloidosis With Polyneuropthy | Active, Not Recruiting |