An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
Summary
Primary Objective: To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks (Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein cholesterol (LDL-C) levels in participants with heterozygous familial hypercholesterolemia (heFH) 8 to 17 years of age on optimal stable daily dose of statin therapy ± other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins. Secondary Objectives: * To evaluate the efficacy of alirocumab versus placebo on LDL-C levels. * To evaluate the effects of alirocumab versus placebo on other lipid parameters. * To evaluate the safety and tolerability of alirocumab in comparison with placebo. * To evaluate the efficacy, safety, and tolerability of alirocumab after open label treatment. * To evaluate the development of anti-alirocumab antibodies.
Timeline
- Start
- 2018-05-31
- Primary completion
- 2021-01-14
- Completion
- 2022-08-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alirocumab | Monoclonal antibody | 40 mg | Subcutaneous |
| Subject | Alirocumab | Monoclonal antibody | 75 mg | Subcutaneous |
| Subject | Alirocumab | Monoclonal antibody | 150 mg | Subcutaneous |
| Subject | Alirocumab | Monoclonal antibody | 300 mg | Subcutaneous |
| Background | Atorvastatin | Small molecule | — | Oral |
| Background | Cholestyramine | Other / unclassified | — | Oral |
| Background | Ezetimibe | Unknown | — | Oral |
| Background | Fenofibrate | Small molecule | — | Oral |
| Background | Fluvastatin | Other / unclassified | — | Oral |
| Background | Lovastatin | Small molecule | — | Oral |
| Background | Niacin | Small molecule | — | Oral |
| Background | Omega-3 fatty acids | Unknown | — | Oral |
| Background | Pravastatin | Small molecule | — | Oral |
| Background | Rosuvastatin | Small molecule | — | Oral |
| Background | Simvastatin | Small molecule | — | Oral |