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Familial chylomicronemia syndrome

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for familial chylomicronemia syndrome

5 trials tracked for familial chylomicronemia syndrome: 4 in phase 3 or 4 and 2 with published results. The most-cited published study has 673 citations.

5Trials tracked
4Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 4 Phase 2 1
  1. Phase 3 The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome Completed · 673 cited
  2. Phase 3 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) Completed · 239 cited
  3. Phase 3 The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome Completed
  4. Phase 3 A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome Completed
  5. Phase 2 Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) Completed

Showing the 5 most-cited and recently-updated of 5 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Familial chylomicronemia syndrome: what the trials found

Plozasiran demonstrated significant reductions in fasting serum triglycerides (TG) at month 10 (-85.7%, -89.5%, and 38.8%; p=0.0022) and when averaged over months 10 and 12 (-82.1%, -86.6%, and 49.5%; p=0.0055). Additionally, it significantly reduced fasting serum Apolipoprotein C3 (APOC3) at month 10 (-92.55%, -91.83%, and 19.91%; p=0.0008).

Volanesorsen showed significant reductions in fasting triglycerides by month 3, with a mean percent change of -76.5% and -49.2% across different reported cohorts [2, 4]. In one trial, Volanesorsen achieved an absolute reduction in fasting TG of -1712 mg/dL (p<0.0001) and a treatment response rate where participants reached <750 mg/dL at month 3 (p=0.0001) 2.

Olezarsen significantly reduced fasting triglycerides at both month 6 (-10.85% and -31.99%; p=0.0009) and month 12 (-22.92% and -38.50%; p=0.0044). It also demonstrated significant reductions in Apolipoprotein C-III (apoC-III) at both time points 3.

Recent results — preliminary, needs further review

  • AKCEA-ANGPTL3-LRx showed a -32.83% reduction in fasting triglycerides and a -51.249% reduction in Angiopoietin-like 3 (ANGPTL3) at month 3, though these results are not yet corroborated.

For the clinician treating this condition

  • Plozasiran provides significant reductions in both fasting triglycerides and APOC3 levels 1.
  • Volanesorsen is associated with substantial decreases in fasting triglyceride levels and high rates of achieving target TG thresholds [2, 4].
  • Olezarsen effectively reduces fasting triglycerides and apoC-III levels over a 12-month period 3.

AI synthesis of 3 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

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