Prevention & lipidsRandomised controlled trial
Longer-Term Efficacy and Safety of Evinacumab in Patients With Refractory Hypercholesterolemia
- Randomised controlled trial (open-label treatment period)
- double-blind treatment period
- placebo-controlled
- 48-week open-label treatment period
- multicentre (85 sites, 20 countries)
Authors' conclusion
In patients with refractory hypercholesterolemia, evinacumab provided sustained reductions in LDL-C level and was generally well tolerated.
Study context
- Population
- Patients with refractory primary hypercholesterolemia (heterozygous familial hypercholesterolemia or established clinical atherosclerotic cardiovascular disease (ASCVD) without familial hypercholesterolemia), refractory to maximally tolerated lipid-lowering therapies (LLTs)88 (91.7%) completed the open-label treatment period (OLTP)
- Participants
- 96 patients
- Primary endpoint
- Change in LDL-C level from baseline
- Follow-up
- Week 72 (end of the OLTP)
Primary result
As reported in the abstract
LDL-C change from baseline · week 72
Evinacumab 15 mg/kg
45.5%
mean reduction · SD 28.7%
Further findings
| Outcome | Values |
|---|---|
| Other lipid/lipoprotein parameters, change from baseline · week 72 | Evinacumab 15 mg/kg Apolipoprotein B, mean (SD) reduction 38.0% [22.1%] · Non-HDL-C, mean (SD) reduction 48.4% [23.2%] · Total cholesterol, mean (SD) reduction 42.6% [17.5%] · Fasting TG, median (IQR) reduction 57.2% [65.4%-44.4%] |
Safety
Treatment-emergent adverse events (TEAEs)
- TEAEs, 78 of 96 patients
- 81.3%
Serious TEAEs
- Serious TEAEs, 9 of 96 patients
- 9.4%
- all considered unrelated to study treatment
Original publication
Longer-Term Efficacy and Safety of Evinacumab in Patients With Refractory Hypercholesterolemia. JAMA Cardiol. Published 1 November 2023. doi:10.1001/jamacardio.2023.2921
Summarised from the published abstract; every number is checked against it