Phase 3
N=58
Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities
Homozygous Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT01588496 ↗Enrolled (actual)
58
Serious AEs
0.0%
Results posted
Oct 2015
Primary outcome: Primary: Part A: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 — -16.5 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Evolocumab (Biological); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Jan 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 |
-16.5 | — |
| PRIMARY Part B: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 |
7.88; -23.05 | <0.001 sig |
| SECONDARY Part A: Change From Baseline in LDL-C at Week 12 |
-70.6 | — |
| SECONDARY Part A: Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 12 |
-16.6 | — |
| SECONDARY Part A: Percent Change From Baseline in Apolipoprotein B at Week 12 |
-14.9 | — |
| SECONDARY Part A: Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12 |
-18.319 | — |
| SECONDARY Part A: Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 12 |
-15.65 | — |
| SECONDARY Part A: Percentage of Participants With 15% or Greater Reduction in LDL-C From Baseline at Week 12 |
50.0 | — |
| SECONDARY Part A: Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) at Week 12 |
-151.3 | — |
| SECONDARY Part B: Percent Change From Baseline in LDL-C at the Mean of Weeks 6 and 12 |
4.22; -25.56 | <0.001 sig |
| SECONDARY Part B: Percent Change From Baseline in Apolipoprotein B at Week 12 |
3.97; -19.17 | <0.001 sig |
| SECONDARY Part B: Percent Change From Baseline in Apolipoprotein B at the Mean of Weeks 6 and 12 |
2.65; -20.24 | <0.001 sig |
| SECONDARY Part B: Percent Change From Baseline in Lipoprotein (a) at Week 12 |
2.43; -9.40 | 0.088 |
| SECONDARY Part B: Percent Change From Baseline in Lipoprotein (a) at the Mean of Weeks 6 and 12 |
-1.43; -12.71 | 0.088 |
Summary
A study to determine the safety, tolerability, and efficacy of evolocumab (AMG 145) in patients with homozygous familial hypercholesterolemia (HoFH).
Eligibility Criteria
Inclusion Criteria
- Males and females ≥ 12 to ≤ 80 years of age
- Diagnosis of homozygous familial hypercholesterolemia
- Stable lipid-lowering therapies for at least 4 weeks
- LDL cholesterol ≥ 130 mg/dl (3.4 mmol/L)
- Triglyceride ≤ 400 mg/dL (4.5 mmol/L)
- Bodyweight of ≥ 40 kg at screening.
Exclusion Criteria
- LDL or plasma apheresis within 8 weeks prior to randomization
- New York Heart Association (NYHA) class III or IV or last known left ventricular ejection fraction < 30%
- Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 3 months of randomization
- Planned cardiac surgery or revascularization
- Uncontrolled cardiac arrhythmia
- Uncontrolled hypertension
Data sourced from ClinicalTrials.gov (NCT01588496). 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.