At a glance
ClinicalIndex Comparison Record- ✓Diagnosis of homozygous familial hypercholesterolemia (HoFH) confirmed by genotype (homozygous/compound heterozygous LDLR, ApoB, PCSK9, or LDLRAP1 mutations; double heterozygous mutations across genes) OR clinical criteria (untreated TC >500 mg/dL with TG <300 mg/dL AND parental history or xanthomas before age 10)
- ✓Receiving stable statin dose at screening, or documented as ineffective/intolerant
- ✓If on LDL apheresis, must have started ≥3 months before screening and maintained stable weekly or every-2-week schedule for ≥8 weeks
- ✓LDL-C level ≥70 mg/dL at screening
- ✕Documented null mutation in both LDLR alleles
- ✕Use of PCSK9 inhibitor within 10 weeks of screening
- ✕Background lipid-modifying therapy not stable for required duration: statin/other agents ≥4 weeks; fibrates ≥6 weeks; mipomersen ≥24 weeks; lomitapide ≥12 weeks
- ✕Nutraceuticals or OTC lipid-affecting therapies not stable for ≥4 weeks prior to screening or between screening and randomization
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia
In Brief
A Phase 3 clinical trial evaluating Alirocumab and Placebo for Homozygous Familial Hypercholesterolemia. Completed, enrolled 69 participants across 28 sites in 13 countries.
Detailed Summary
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment. The secondary objectives of the study are: * To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein \[Apo\] A-1 and B, non-high-density lipoprotein cholesterol \[non-HDL-C\], total-cholesterol \[TC\], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides \[TG\]) in participants with HoFH * To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH * To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH * To assess the potential development of anti-drug (alirocumab) antibodies
Study Details
Timeline
Interventions
Alirocumab SC Q2W
Matching placebo SC Q2W