An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Summary
Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams \[mg\] depending on body weight \[BW\]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments. Secondary Objectives: * To evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on LDL-C levels. * To evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein B \[Apo B\], non-high density lipoprotein cholesterol \[non-HDL-C\], total cholesterol \[Total-C\], high density lipoprotein cholesterol \[HDL-C\], lipoprotein a \[Lp (a)\], triglycerides \[TG\], apolipoprotein A-1 \[Apo A-1\] levels) after 12, 24, and 48 weeks of treatment. * To evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.
Timeline
- Start
- 2018-08-31
- Primary completion
- 2020-02-17
- Completion
- 2020-02-17
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alirocumab | Monoclonal antibody | 75 mg | Subcutaneous |
| Subject | Alirocumab | Monoclonal antibody | 150 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 |