Phase 3
N=331
Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2
Hyperlipidemia
Bottom Line
View on ClinicalTrials.gov: NCT01763918 ↗Enrolled (actual)
331
Serious AEs
3.7%
Results posted
Dec 2015
Primary outcome: Primary: Percent Change From Baseline in LDL-C at Week 12 — -2.02; 5.53; -61.25; -55.74 percent change — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Evolocumab (Biological); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Nov 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in LDL-C at Week 12 |
-2.02; 5.53; -61.25; -55.74 | <0.001 sig |
| PRIMARY Percent Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 |
-1.08; 2.30; -61.23; -63.25 | <0.001 sig |
| SECONDARY Change From Baseline in LDL-C at the Mean of Weeks 10 and 12 |
-6.5; -1.3; -101.7; -98.8 | <0.001 sig |
| SECONDARY Change From Baseline in LDL-C at Week 12 |
-8.5; 4.1; -101.3; -87.2 | <0.001 sig |
| SECONDARY Percentage of Participants With Mean LDL-C at Weeks 10 and 12 of Less Than 70 mg/dL (1.8 mmol/L) |
1.9; 1.9; 67.0; 80.4 | <0.001 sig |
| SECONDARY Percentage of Participants With LDL-C < 70 mg/dL (1.8 mmol/L) at Week 12 |
2.0; 2.2; 68.3; 63.1 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at the Mean of Weeks 10 and 12 |
0.21; 2.72; -55.79; -57.28 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Non-HDL-C at Week 12 |
-1.39; 5.29; -56.19; -49.67 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B at the Mean of Weeks 10 and 12 |
-0.19; 2.21; -49.58; -52.76 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B at Week 12 |
-0.67; 4.60; -49.75; -44.81 | <0.001 sig |
| SECONDARY Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at the Mean of Weeks 10 and 12 |
0.86; 4.14; -45.74; -45.02 | <0.001 sig |
| SECONDARY Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12 |
0.12; 7.11; -45.95; -38.32 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at the Mean of Weeks 10 and 12 |
0.78; 1.65; -52.39; -53.91 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 12 |
1.54; 4.23; -52.74; -45.31 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at the Mean of Weeks 10 and 12 |
7.34; 5.35; -24.03; -25.65 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) at Week 12 |
8.68; 6.69; -22.89; -21.55 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Triglycerides at the Mean of Weeks 10 and 12 |
9.09; 7.49; -13.27; -9.25 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Triglycerides at Week 12 |
3.50; 6.43; -16.09; -5.13 | <0.001 sig |
| SECONDARY Percent Change From Baseline in HDL-C at the Mean of Weeks 10 and 12 |
-0.45; -2.86; 7.93; 6.62 | <0.001 sig |
| SECONDARY Percent Change From Baseline in HDL-C at Week 12 |
-1.15; -3.73; 8.05; 5.35 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Very Low-Density Lipoprotein Cholesterol (VLDL-C) at the Mean of Weeks 10 and 12 |
8.66; 6.34; -13.97; -9.20 | <0.001 sig |
| SECONDARY Percent Change From Baseline in VLDL-C at Week 12 |
3.73; 4.10; -17.25; -5.06 | <0.001 sig |
Summary
The primary objective was to evaluate the effect of 12 weeks of evolocumab subcutaneously once every 2 weeks (Q2W) and once monthly (QM), compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in adults with heterozygous familial hypercholesterolemia (HeFH).
Eligibility Criteria
Inclusion Criteria
- Male or female ≥ 18 to ≤ 80 years of age
- Diagnosis of heterozygous familial hypercholesterolemia
- On a stable dose of an approved statin and lipid regulating medication
- Fasting LDL-C ≥ 100 mg/dL (2.6 mmol/L)
- Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Exclusion Criteria
- Homozygous familial hypercholesterolemia
- LDL or plasma apheresis
- New York Heart Association (NYHA) III or IV heart failure
- Uncontrolled cardiac arrhythmia
- Uncontrolled hypertension
- Type 1 diabetes, poorly controlled type 2 diabetes
- Uncontrolled hypothyroidism or hyperthyroidism
Data sourced from ClinicalTrials.gov (NCT01763918). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.