FCS BALANCE Trial
Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome (FCS)
A phase 3, double-blind, placebo-controlled trial
Objective:
To assess the efficacy and safety of olezarsen (given once every 4 weeks) in reducing fasting triglyceride level in patients with familial chylomicronemia syndrome.
66 Patients
Inclusion Criteria:
- Aged 18 years or above
- Genetically confirmed familial chylomicronemia syndrome
- Fasting TG ≥ 880 mg/dL and history of pancreatitis
Exclusion Criteria:
- Acute coronary syndrome within 6 months
- Major surgery within the last 3 months
Groups:
- A: Olezarsen 80 mg (n = 22)
- B: Olezarsen 50 mg (n = 21)
- C: Placebo (n = 23)
Primary Outcomes:
- Mean % change in Triglyceride level at 6 months
- (A vs C): -43.5% (P < 0.001)
- (B vs C): -22.4% (P < 0.001)
Secondary Outcomes:
- Mean % change in apolipoprotein C-III level at 6 months
- (A vs C): -73.7% (95% CI: -94.6 to -52.8)
- (B vs C): -65.5% (95% CI: -82.6 to -48.3)
Conclusion:
In adults with familial chylomicronemia syndrome, 80 mg of olearsen given subcutaneously once every 4 weeks reduced fasting triglyceride levels at 6 months significantly more than placebo.