Lipids & metabolic liver disease
Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, non-inferiority trial
Primary result · Percent change in LDL cholesterolResult — go to the resultMean difference 5.4 % 95% CI −0.2 to 11.1Authors' conclusion
The LDL cholesterol response was highly variable, but generally similar in patients treated with both lerodalcibep and evolocumab. Importantly, the study showed the inability to predict response based on genotyping, reinforcing the rationale for PCSK9 inhibition in all patients with homozygous familial hypercholesterolemia and continuing its use in responders.
Study context
- Population
- Patients aged ≥10 years with genetically confirmed homozygous familial hypercholesterolaemia; mean age 28.7 years, 30% paediatric, mean baseline LDL cholesterol 10.59 mmol/L82 screened
- Participants
- 66 patients
- Comparison
- Monthly lerodalcibep 300 mg vs monthly evolocumab 420 mg
- Primary endpoint
- Percent change from baseline (day 1 of period A) in LDL cholesterol concentration
- Follow-up
- Week 24 for period A and B
Methodological detailsphase 3, open-label, crossover, non-inferiority, two 24-week treatment periods separated by an 8-week washout, 12 lipid clinics in six countries, Enrolled Nov 11, 2019, to July 2, 2021; final study visit Aug 8, 2022
- Phase 3
- Open-label
- Crossover
- Non-inferiority
- Two 24-week treatment periods separated by an 8-week washout
- 12 lipid clinics in six countries
- Enrolled Nov 11, 2019, to July 2, 2021; final study visit Aug 8, 2022
Primary result
Percent change in LDL cholesterol · week 24
Mean difference
5.4 %
95% CI −0.2 to 11.1
Change by group
Lerodalcibep
−4.9
mean · SE 3.5 · %
Evolocumab
−10.3
mean · SE 3.5 · %
As reported in the abstract
Further findings
%
| Outcome | Lerodalcibep | Evolocumab |
|---|---|---|
| LDL cholesterol response averaged across all monthly visits | −9.1 (SE 3.2) | −10.8 (SE 3.2) |
Predictors of response
- Genotyping and free PCSK9 suppression were not predictive of response
- LDL cholesterol response varied considerably but was generally similar in the same patients with both drugs
Safety
Injection site reactions
Denominator Lerodalcibep 65 · Evolocumab 62
Tolerability and serious adverse events
- Both drugs well tolerated; no treatment-related serious adverse events
- 0
Original publication
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