Phase 2
N=23
A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene
Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT01604824 ↗Enrolled (actual)
23
Serious AEs
9.1%
Results posted
Jun 2020
Primary outcome: Primary: Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15 — -62.48; -8.77; -48.21; -4.93 percent change — p== 0.0009
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Alirocumab (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Jun 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15 |
-62.48; -8.77; -48.21; -4.93 | = 0.0009 sig |
| SECONDARY Percent Change in Apolipoprotein (Apo) B100 From Baseline to Day 15 |
-53.33; -3.78; -47.73; -3.09 | = 0.0021 sig |
| SECONDARY Percent Change in Non High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Day 15 |
-56.87; -7.50; -44.40; -4.04 | = 0.0016 sig |
| SECONDARY Percent Change in Total Cholesterol (Total-C) From Baseline to Day 15 |
-36.94; -6.18; -29.40; -7.18 | = 0.0017 sig |
| SECONDARY Percent Change in Apolipoprotein (Apo) B100/ ApoA-1 Ratio From Baseline to Day 15 |
-55.26; -5.53; -48.34; 0.99 | = 0.0005 sig |
Summary
The primary objective of the study is to assess the pharmacodynamic (PD) effect of alirocumab on serum low density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneous (SC) administered alirocumab in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutation (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with loss-of-function mutation (LOFm) in 1 or more alleles of the apolipoprotein (ApoB) gene.
Eligibility Criteria
Inclusion Criteria
Inclusion criteria include, but are not limited to the following:
- Between the ages of 18 and 70 years, inclusive
- A history of molecularly confirmed PCSK9 GOFm for cohort 1 and a history of molecularly confirmed PCSK9 GOFm or ApoB LOFm
- Plasma LDL-Cholesterol levels ≥70 mg/dL at the screening visit on a lipid-lowering therapy (LLT) regimen stable for at least 28 days
Exclusion Criteria
Exclusion criteria include, but are not limited to the following:
- Serum triglycerides >350 mg/dL at the screening visit
- Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus
- Pregnant or breast-feeding women.
- Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study
Data sourced from ClinicalTrials.gov (NCT01604824). 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.