Phase 2
N=168
Reduction of Low-Density Lipoprotein Cholesterol (LDL-C) With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study
Hypercholesterolemia, Familial
Bottom Line
View on ClinicalTrials.gov: NCT01375751 ↗Enrolled (actual)
168
Serious AEs
1.2%
Results posted
Sep 2015
Primary outcome: Primary: 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
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Evolocumab (Biological); Placebo (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- May 2012
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 |
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).
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.