Familial Chylomicronemia Syndrome
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CLINICAL_TRIALS other · 2026-03-03Clinical Trial Update: Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCS
ClinicalTrials.gov update describes the treatment protocol for the investigational drug Plozasiran in adults with high-risk severe hypertrig…
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome [Phase 3]
Phase 3 study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese adults with Familial Chylomicronemia Syndrome has completed, and res…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: CS-121 APOC3 Base Editing in FCS [EARLY_Phase 1]
Early-phase trial update for CS-121 APOC3 base editing for familial chylomicronemia syndrome, currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-02-04Clinical Trial Update: Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) [Phase 3]
Phase 3 study results have been posted for ARO-APOC3 (Plozasiran) in adults with Familial chylomicronemia syndrome; the trial is currently a…
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CLINICAL_TRIALS trial_result · 2026-02-04Clinical Trial Update: Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) [Phase 3]
Phase 3 clinical trial of ARO-APOC3 (Plozasiran) in adults with Familial Chylomicronemia Syndrome has completed, with results posted.
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CLINICAL_TRIALS research · 2026-01-28Clinical Trial Update: CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia [EARLY_Phase 1]
CS-121 APOC3 base editing for hyperchylomicronemia in children and adolescents is in EARLY Phase 1 and currently recruiting.
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CLINICAL_TRIALS trial_result · 2025-12-30Clinical Trial Update: Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects [Phase 1, Phase 2]
RN0361 is being studied in healthy adults and adults with hypertriglyceridemia (including familial chylomicronemia syndrome) in Phase 1 and …
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS) [Phase 1]
Phase 1 study evaluating ARO-APOC3 in healthy volunteers and patients with hypertriglyceridemia or familial chylomicronemia syndrome has com…
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CLINICAL_TRIALS research · 2025-12-12Clinical Trial Update: A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen [Phase 3]
Phase 3 trial update for Olezarsen in adults with Familial chylomicronemia syndrome (FCS) who were previously treated with Volanesorsen; the…
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CLINICAL_TRIALS research · 2025-12-12Clinical Trial Update: A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) [Phase 3]
A Phase 3 trial update for olezarsen (AKCEA-APOCIII-LRx) in Familial Chylomicronemia Syndrome indicates the study is active but not currentl…
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CLINICAL_TRIALS trial_result · 2025-03-06Clinical Trial Update: A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) [Phase 3]
Phase 3 trial of olezarsen in familial chylomicronemia syndrome has completed and results have been posted.
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CLINICAL_TRIALS approval · 2024-12-27Clinical Trial Update: Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
This update notes that Olezarsen has been approved for marketing under an Early Access Program for patients with Familial Chylomicronemia Sy…
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FDA approval · 2024-12-19FDA Approved: TRYNGOLZA — Original Application (Ionis Pharmaceuticals, Inc.)
FDA approves TRYNGOLZA (olesarsen sodium) from Ionis Pharmaceuticals as a new molecular entity for the treatment of severe hypertriglyceride…
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CLINICAL_TRIALS trial_result · 2022-04-13Clinical Trial Update: The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome [Phase 3]
Phase 3 trial of volanesorsen in familial chylomicronemia syndrome completed with posted results.
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CLINICAL_TRIALS trial_result · 2021-08-26Clinical Trial Update: The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome [Phase 3]
Phase 3 open-label study of volanesorsen in familial chylomicronemia syndrome has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-01-07Clinical Trial Update: Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) [Phase 2]
Phase 2 trial update for AKCEA-ANGPTL3-LRx (ISIS 703802) in Familial Chylomicronemia Syndrome (and related lipoprotein lipase deficiency) ha…
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CLINICAL_TRIALS trial_result · 2016-11-15Clinical Trial Update: Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome. [Phase 3]
Extension of a Phase 3 randomized, double-blind, placebo-controlled study of LCQ908 in familial chylomicronemia syndrome was terminated, wit…
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CLINICAL_TRIALS trial_result · 2016-07-21Clinical Trial Update: Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia [Phase 2]
A Phase 2 pilot study evaluating CAT-2003 for familial chylomicronemia syndrome and related lipid disorders has completed.
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CLINICAL_TRIALS trial_result · 2015-06-03Clinical Trial Update: A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome [Phase 3]
A Phase 3 randomized, double-blind, placebo-controlled trial evaluating LCQ908 in familial chylomicronemia syndrome has completed with resul…
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CLINICAL_TRIALS trial_result · 2011-09-29Clinical Trial Update: Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects [Phase 2, Phase 3]
Completed Phase 2/3 trial evaluating the efficacy and safety of LPL S447X gene therapy expressed by an adeno-associated viral vector in indi…