Phase 2
Completed N=168
Reduction of Low-Density Lipoprotein Cholesterol (LDL-C) With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study
Hypercholesterolemia, Familial
Source: ClinicalTrials.gov NCT01375751 ↗
Enrolled (actual)
168
Serious AEs
1.2%
Results posted
Sep 2015
Primary outcomePrimary: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12 — 1.12; -42.70; -55.24 percent change — p=<0.001
Summary
The primary objective of this study was to evaluate the effect of 12 weeks of subcutaneous evolocumab (AMG 145), compared with placebo, on percent change from baseline in LDL-C in adults with heterozygous familial hypercholesterolemia (HeFH).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12 |
1.12; -42.70; -55.24 | <0.001 sig |
| SECONDARY Absolute Change From Baseline in LDL-C at Week 12 |
4.2; -61.3; -80.5 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (HDL-C) at Week 12 |
2.48; -39.31; -50.98 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B at Week 12 |
2.86; -31.89; -43.34 | <0.001 sig |
| SECONDARY Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12 |
2.98; -33.69; -42.03 | <0.001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B /Apolipoprotein A-1 Ratio at Week 12 |
-4.10; -38.02; -48.74 | <0.001 sig |
Eligibility Criteria
Inclusion Criteria
- Male or female ≥ 18 to ≤ 75 years of age
- Diagnosis of heterozygous familial hypercholesterolemia by having met the diagnostic criteria outlined by the Simon Broome Register Group (Scientific Steering Committee 1991)
- On an approved statin, with or without ezetimibe, with stable dose(s) for at least 4 weeks
- Fasting Low-Density Lipoprotein Cholesterol (LDL-C) ≥ 100 mg/dL
- Fasting triglycerides ≤ 400 mg/dL
Exclusion Criteria
- Homozygous familial hypercholesterolemia
- Low-Density Lipoprotein (LDL) or plasma apheresis within 12 months prior to randomization
- New York Heart Association (NYHA) III or IV heart failure, or known left ventricular ejection fraction 8.5%)
- Uncontrolled hypertension
Data sourced from ClinicalTrials.gov (NCT01375751). 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. Informational only — not medical advice.