7 completed, 3 active not recruiting, 1 approved for marketing, 1 recruiting. 7 Phase 3, 2 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 7 | Completed 4, Active not recruiting 2, Recruiting 1 |
| Phase 2 | 2 | Active not recruiting 1, Completed 1 |
| Phase 1 | 1 | Active not recruiting 1 |
Follow Familial Chylomicronemia Syndrome: new trials by email Literature Review of Familial Chylomicronemia Syndrome trials Research Report on Familial Chylomicronemia Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants Olezarsen - Familial Chylomicronemia Syndrome | Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc. | 12 | 2026-07-31 | 2026-10-08 |
| Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects RN0361 - Hypertriglyceridemia, Familial Chylomicronemia Syndrome | Phase 1 / Phase 2 | Active not recruiting | Ikaria Bioscience Pty Ltd | 108 | 2024-09-30 | 2025-12-30 |
| A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome Plozasiran, Placebon - Familial Chylomicronemia Syndrome | Phase 3 | Completed | Visirna Therapeutics HK Limited | 37 | 2023-07-10 | 2026-02-27 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen Olezarsen - Familial Chylomicronemia Syndrome | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 24 | 2022-02-25 | 2025-12-12 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) Olezarsen - Familial Chylomicronemia Syndrome | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 60 | 2021-11-18 | 2025-12-12 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) Olezarsen, Placebo - Familial Chylomicronemia Syndrome | Phase 3 | Completed | Ionis Pharmaceuticals, Inc. | 66 | 2020-11-18 | 2025-03-06 |
| InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia observational : questionnaries for assessment of health burden - Familial Chylomicronemia Syndrome, Multifactorial Chylomicronemia Syndrome | Completed | New French Society of Atherosclerosis | 52 | 2018-09-19 | 2020-01-10 | |
| Medical Complications in Familial and Multifactorial Chylomicronaemia Syndromes Familial Chylomicronemia Syndrome, Multifactorial Chylomicronemia Syndrome | Completed | Hospices Civils de Lyon | 140 | 2018-03-01 | 2019-04-11 | |
| Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) AKCEA-ANGPTL3-LRx - Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | Phase 2 | Completed | Akcea Therapeutics | 3 | 2017-12-21 | 2021-01-07 |
| The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome Volanesorsen - Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | Phase 3 | Completed | Akcea Therapeutics | 68 | 2015-12-23 | 2021-08-26 |
| The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome Volanesorsen, Placebo - Familial Chylomicronemia Syndrome | Phase 3 | Completed | Ionis Pharmaceuticals, Inc. | 67 | 2014-12-01 | 2022-04-13 |
| Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) Olezarsen - Familial Chylomicronemia Syndrome | Approved for marketing | Ionis Pharmaceuticals, Inc. | 2024-12-27 |
Familial hyperchylomicronemia syndrome (12) · Hyperlipoproteinemia Type I (3) · Dyslipidemias (2) · Genetic Diseases, Inborn (2) · Hyperlipidemias (2) · Hyperlipoproteinemias (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Hypertriglyceridemia (1)
Nutritional and Metabolic Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Dyslipidemias (2) · Hyperlipidemias (2) · Lipid Metabolism Disorders (2) · Metabolic Diseases (2) · Genetic Diseases, Inborn (1) · Hyperlipoproteinemias (1) · Lipid Metabolism, Inborn Errors (1) · Metabolism, Inborn Errors (1)
olezarsen (5) · ISIS 304801 (2) · Apolipoprotein C-III (1) · IONIS-ANGPTL3-LRx (1) · Saline Solution (1) · plozasiran (1) · vupanorsen (1)
Ionis Pharmaceuticals, Inc. (6) · Akcea Therapeutics (2) · Hospices Civils de Lyon (1) · Ikaria Bioscience Pty Ltd (1) · New French Society of Atherosclerosis (1) · Visirna Therapeutics HK Limited (1)
United States (8) · Canada (6) · France (6) · Italy (4) · Netherlands (4) · Spain (4) · United Kingdom (4) · Sweden (3) · Brazil (2) · Germany (2) · Israel (2) · Norway (2) · Portugal (2) · Slovakia (2) · South Africa (2)
Philippe Moulin (1) · Philippe Moulin, MD (1)
PMID 27271183, PMID 29748148, PMID 31390500, PMID 33221907, PMID 37164837, PMID 38587247, PMID 41545246
2014 (1), 2015 (1), 2023 (1)