Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities
Phase 2
Completed
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Biological: EvolocumabDrug: Placebo
- Registration Number
- NCT01588496
- Lead Sponsor
- Amgen
- Brief Summary
A study to determine the safety, tolerability, and efficacy of evolocumab (AMG 145) in patients with homozygous familial hypercholesterolemia (HoFH).
- Detailed Description
Study Masking:
Part A: Open Label Part B: Double Blind
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 58
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part B: Evolocumab Evolocumab Participants received double-blind evolocumab 420 mg subcutaneously once a month for 12 weeks. Part A: Evolocumab Evolocumab Participants received open-label evolocumab 420 mg subcutaneously once a month for 12 weeks. Part B: Placebo Placebo Participants received double-blind placebo subcutaneously once a month for 12 weeks.
- Primary Outcome Measures
Name Time Method Part B: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 Baseline and Week 12 LDL-C was quantified using the ultracentrifugation method.
Part A: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 Baseline and Week 12 LDL-C was quantified using the ultracentrifugation method.
- Secondary Outcome Measures
Name Time Method Part B: Percent Change From Baseline in Lipoprotein (a) at Week 12 Baseline and Week 12 Part B: Percent Change From Baseline in LDL-C at the Mean of Weeks 6 and 12 Baseline and Weeks 6 and 12 LDL-C was quantified using the ultracentrifugation method.
Part B: Percent Change From Baseline in Apolipoprotein B at the Mean of Weeks 6 and 12 Baseline and Weeks 6 and 12 Part A: Change From Baseline in LDL-C at Week 12 Baseline and Week 12 LDL-C was quantified using the ultracentrifugation method.
Part A: Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Week 12 Baseline and Week 12 Part A: Percent Change From Baseline in Apolipoprotein B at Week 12 Baseline and Week 12 Part A: Percent Change From Baseline in the Total Cholesterol/HDL-C Ratio at Week 12 Baseline and Week 12 Part A: Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio at Week 12 Baseline and Week 12 Part A: Percentage of Participants With 15% or Greater Reduction in LDL-C From Baseline at Week 12 Baseline and Week 12 LDL-C was quantified using the ultracentrifugation method.
Part B: Percent Change From Baseline in Apolipoprotein B at Week 12 Baseline and Week 12 Part A: Change From Baseline in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) at Week 12 Baseline and Week 12 Part B: Percent Change From Baseline in Lipoprotein (a) at the Mean of Weeks 6 and 12 Baseline and Weeks 6 and 12
Trial Locations
- Locations (1)
Research Site
🇪🇸Madrid, Spain