Prevention & lipidsRandomised controlled trial
Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome
- Randomised controlled trial
- phase 3
- double-blind
- placebo-controlled
Authors' conclusion
In patients with familial chylomicronemia syndrome, olezarsen may represent a new therapy to reduce plasma triglyceride levels.
Study context
- Population
- Patients with genetically identified familial chylomicronemia syndrome
- Participants
- 66 patients
- Comparison
- Olezarsen 80 mg or 50 mg vs placebo
- Olezarsen 80 mgn = 22
- Olezarsen 50 mgn = 21
- Placebon = 23reference
- Primary endpoint
- Percent change in fasting triglyceride level, 80 mg vs placebo, then (if significant) 50 mg vs placebo
- Follow-up
- 6 months
Primary result
As reported in the abstract
Percent change in fasting triglyceride level
Each group vs placeboDifference, percentage points (95% CI)P value
Olezarsen 80 mg
−43.5(−69.1 to −17.9)
P < 0.001Olezarsen 50 mg
−22.4(−47.2 to 2.5)
P = 0.08Secondary outcomes
| Outcome | difference (percentage points) (95% CI) | |
|---|---|---|
| Mean percent change in apolipoprotein C-III level · 6 months — Olezarsen 80 mg vs Placebo | −73.7 (−94.6 to −52.8) | |
| Mean percent change in apolipoprotein C-III level · 6 months — Olezarsen 50 mg vs Placebo | −65.5 (−82.6 to −48.3) | |
| −100−50050Difference, percentage points (linear scale) |
events
| Outcome | Olezarsen 80 mg | Olezarsen 50 mg | Placebo | rate ratio (95% CI) | |
|---|---|---|---|---|---|
| Adjudicated acute pancreatitis episodes · 53 weeks · pooled olezarsen groups | 1 | 1 | 11 | 0.12 (0.02–0.66) | |
| 0.050.21520Rate ratio (log scale) |
Safety
| Outcome | Olezarsen 80 mg | Olezarsen 50 mg | Placebo |
|---|---|---|---|
| Moderate drug-related adverse events (investigator-assessed) | 4 events | Not reported in the abstract | Not reported in the abstract |
Original publication
Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. Published 7 April 2024. doi:10.1056/nejmoa2400201
Summarised from the published abstract; every number is checked against it