8 completed, 3 active not recruiting, 2 recruiting, 2 terminated. 9 Phase 3, 3 Phase 2, 1 Early Phase 1, 1 Phase 1.
Follow Familial hyperchylomicronemia syndrome: new trials by email Literature Review of Familial hyperchylomicronemia syndrome trials Research Report on Familial hyperchylomicronemia 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 |
| CS-121 APOC3 Base Editing in FCS CS-121 - Familial Chylomicronemia Syndrome (FCS) | Early Phase 1 | Recruiting | CorrectSequence Therapeutics Co., Ltd | 15 | 2025-10-15 | 2026-02-11 |
| 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 |
| Phase 2 Study of Orlistat and SLx-4090 for the Treatment of Type 1 Hyperlipoproteinemia SLx-4090 placebo, Orlistat Placebo, Orlistat - Type 1 Hyperlipoproteinemia | Phase 2 | Terminated | University of Texas Southwestern Medical Center | 5 | 2015-11-01 | 2021-08-19 |
| 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 |
| Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome. LCQ908 - Familial Chylomicronemia Syndrome (FCS) (HLP Type I) | Phase 3 | Terminated | Novartis Pharmaceuticals | 38 | 2013-02-01 | 2016-11-15 |
| A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome LCQ908, Placebo - Familial Chylomicronemia Syndrome (FCS) | Phase 3 | Completed | Novartis Pharmaceuticals | 45 | 2012-07-01 | 2015-06-03 |
| Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) Volanesorsen - Familial Chylomicronemia | No longer available | Akcea Therapeutics | 2021-09-16 | |||
| Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) Olezarsen - Familial Chylomicronemia Syndrome | Approved for marketing | Ionis Pharmaceuticals, Inc. | 2024-12-27 |
Hyperlipoproteinemia Type I (4) · Hyperlipoproteinemias (4) · Dyslipidemias (3) · Genetic Diseases, Inborn (3) · Hyperlipidemias (3) · Hypertriglyceridemia (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Pancreatitis (1)
Nutritional and Metabolic Diseases (8) · Dyslipidemias (5) · Hyperlipidemias (5) · Lipid Metabolism Disorders (5) · Metabolic Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Digestive System Diseases (1) · Genetic Diseases, Inborn (1) · Hyperlipoproteinemias (1) · Lipid Metabolism, Inborn Errors (1) · Metabolism, Inborn Errors (1) · Pancreatic Diseases (1)
olezarsen (5) · ISIS 304801 (3) · pradigastat (2) · 6-(4'-trifluoromethyl-6-methoxybiphenyl-2-ylcarboxamido)-1,2,3,4-tetrahydroisoquinoline-2-carboxylic acid phenyl ester (1) · Apolipoprotein C-III (1) · IONIS-ANGPTL3-LRx (1) · Orlistat (1) · Saline Solution (1) · plozasiran (1) · vupanorsen (1)
Ionis Pharmaceuticals, Inc. (6) · Akcea Therapeutics (3) · Novartis Pharmaceuticals (2) · CorrectSequence Therapeutics Co., Ltd (1) · Hospices Civils de Lyon (1) · Ikaria Bioscience Pty Ltd (1) · New French Society of Atherosclerosis (1) · University of Texas Southwestern Medical Center (1) · Visirna Therapeutics HK Limited (1)
United States (11) · Canada (8) · France (8) · Netherlands (6) · United Kingdom (6) · Spain (5) · Germany (4) · Italy (4) · South Africa (4) · Sweden (3) · Brazil (2) · China (2) · Israel (2) · Norway (2) · Portugal (2)
Abhimanyu Garg, MD (1) · 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)