Phase 3
N=300
Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
Severe Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT01624142 ↗Enrolled (actual)
300
Serious AEs
28.7%
Results posted
Jan 2019
Primary outcome: Primary: Number of Participants With Adverse Events — 94; 174; 76; 143 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Evolocumab (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- May 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events |
94; 174; 76; 143; 38; 68 | — |
| SECONDARY Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) |
-20.42; -54.95; -18.32; -67.57; -20.28; -55.03 | — |
| SECONDARY Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) |
-19.62; -50.00; -18.17; -59.76; -20.29; -50.61 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) |
-11.11; -20.49; -8.97; -24.42; -9.95; -21.51 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B |
-16.91; -43.84; -16.25; -56.70; -17.57; -43.95 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol/HDL-C Ratio |
-21.32; -43.49; -23.25; -52.38; -23.93; -43.44 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio |
-23.06; -46.79; -21.38; -59.77; -21.53; -47.82 | — |
| SECONDARY Percentage of Participants With a 15% or Greater Reduction in LDL-C |
62.1; 98.9; 51.8; 98.2; 57.4; 97.9 | — |
Summary
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
Eligibility Criteria
Inclusion Criteria
- Participated in Study 20110233 (NCT01588496) or another qualifying evolocumab parent protocol and have a diagnosis of familial hypercholesterolemia.
OR
- Have a diagnosis of familial hypercholesterolemia AND
- Males and females ≥ 12 to ≤ 80 years of age
- Stable low-fat diet and lipid-lowering therapies for at least 4 weeks
- Low-density lipoprotein cholesterol (LDL-C) >= 130 mg/dl (3.4 mmol/L) for subjects without diagnosed coronary heart disease (CHD)/CHD risk equivalent OR LDL-C >= 100 mg/dl (2.6 mmol/L) for subjects with diagnosed CHD or CHD risk equivalent OR apheresis patients have no LDL-C entry requirement
- Fasting triglycerides ≤ 400 mg/dL(4.5 mmol/L)
- Body weight of > 40 kg or greater at screening for subjects less than 18 years of age
Exclusion Criteria
- New York Heart Failure 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 screening
- Planned cardiac surgery or revascularization
- Uncontrolled cardiac arrhythmia
- Uncontrolled hypertension
Data sourced from ClinicalTrials.gov (NCT01624142). 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.