Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH
brief summary
The goal of this clinical trial is to assess the LDL-Cholesterol reductions at Week 12 and Week 24 with monthly dosing of lerodalcibep (Lerochol) 300 mg administered subcutaneously by auto-injector (AI)/pre-filled pen (PFP) compared to placebo (dummy), in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins. The main question\[s\] it aims to answer are: How effective is Lerochol in reducing LDL cholesterol? How well is it tolerated and are there any safety concerns? Researchers will compare Lerochol to placebo (inert or dummy injection solution). Participants will visit the clinic every month for months and be asked to fast overnight, but allowed to drink water, before clinic visits. Undergo physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.
detailed description
This is a randomized, placebo-controlled, double-blind, Phase 3b study to evaluate the efficacy, safety, and tolerability of lerodalcibep 300 mg administered SC QM over a 24-week Treatment Period. Approximately 150 males and females, 6 to 17 years of age, with HeFH and who fulfill the inclusion and exclusion criteria will be enrolled at approximately 30 sites in the United States, Canada, Central and South America, Europe, South Africa, Asia, Australasia, and the Middle East. Patients will be randomized in a 2:1 ratio to lerodalcibep 300 mg (100 patients) or placebo (50 patients) administered SC QM. The study will consist of a Screening Period and a Treatment Period. The total study duration will be up to 35 weeks which includes up to an 11-week Screening Period (which may include up to an 8 week washout) and 24 weeks of study drug treatment
Following randomization and dosing on Day 1, patients will be seen in the clinic QM for 20 weeks and then at 2 week intervals for Weeks 22 and 24. All patients will receive doses of lerodalcibep 300 mg QM or placebo on Day 1 and Weeks 4, 8, 12, 16, and 20.
OBJECTIVES:
The co-primary objectives of this study are to assess the LDL-C reductions at Week 12 and Week 24 with monthly dosing of lerodalcibep 300 mg compared to placebo, in pediatric patients 6 to 17 years of age, with HeFH on a stable diet and maximally tolerated oral LDL C lowering drug therapy.
The secondary objectives of this study are the following:
* To assess the LDL-C-lowering effects of lerodalcibep with LDL-C calculated by Friedewald formula compared to placebo at Week 22 and the mean of Weeks 22 and 24; * To assess the change in LDL-C, measured by preparative ultracentrifugation (PUC), from baseline (Week 0) with lerodalcibep compared to placebo at Week 24; * To assess safety and tolerability of lerodalcibep in pediatric patients with HeFH; * To assess the pharmacodynamic (PD) effects of 300 mg lerodalcibep QM on serum unbound (free) PCSK9 concentrations at Weeks 22 and 24; * To assess the effects of lerodalcibep on serum lipids, including total cholesterol (TC), high density lipoprotein cholesterol (HDL-C), non-HDL-C, very low-density lipoprotein cholesterol (VLDL-C), and triglycerides (TG); * To assess the effects of lerodalcibep on ApoB and lipoprotein (a) (Lp\[a\]) serum concentrations compared to placebo at Weeks 12, 22, and 24, and the mean of Weeks 22 and 24; * To assess the pharmacokinetics (PK) of lerodalcibep and total PCSK9 following 300 mg QM SC doses of lerodalcibep at Weeks 22 (peak post-dose) and 24 (trough post-dose); and * To assess the frequency and level of anti-drug antibodies (ADAs) (immunogenicity) following multiple SC doses of lerodalcibep.
official title
Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy (LIBerate-Kids)