Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents With Familial Hypercholesterolemia

Overview

The goal is to assess the long term efficacy (LDL cholesterol reduction) and safety over 3 years of lerodalcibep (Lerochol) SC 300 mg QM administered by auto-injector (AI)/pre-filled pen (PFP) 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 who completed the 24 week placebo controlled base trial. The main question\[s\] it aims to answer are: How effective is Lerochol in maintaining LDL cholesterol reductions over years? How well is it tolerated and are there any safety concerns? Participants will visit the clinic every month for 3 months and then home dosed with clinic visits every 3 months. They will undergo periodic 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.

STUDY DESIGN AND DURATION: This is an open label Phase 3b study to evaluate the long-term efficacy, safety, and tolerability of lerodalcibep 300 mg administered SC QM (\<31 days) over a 156-week Treatment Period. Approximately 150 males and females 6 to 17 years of age at time of entry in the base LIB003 008 study, with HeFH and who completed the LIB003-008 study at approximately 30 sites in the United States, Canada, Central and South America, Europe, South Africa, Asia, Australasia, and the Middle East may be eligible to continue into this 156-week long term open-label extension with lerodalcibep after signing an independent informed consent form. Patients maintaining their statin with or without ezetimibe and other allowed oral LDL-C-lowering agents will be eligible. Study Procedures Patients who meet all eligibility criteria for the LIB003-016 study may proceed directly to the first dose of lerodalcibep at the final visit of the LIB003-008 study. Patients will receive SC doses, via auto-injector (AI)/prefilled pen (PFP) of lerodalcibep 300 mg QM (31 days) until Week 152. Following administration of the last dose at Week 152, the patients will return 4 weeks later for follow-up at Week 156. Patients may begin the OLE on the final visit of their prior study and/or have up to 28 days to return for Day 1 of the OLE. The period between the base study and LIB003-008 may be extended upon review and approval. Patients will be able to be dosed at home between clinic visits. In addition to a basic lipid profile, LDL-C will be measured by PUC at specified visits during the study. Safety assessments will include adverse events (AEs) and the results from physical examinations, including Tanner staging, skin and Achilles/extensor tendon xanthoma, electrocardiograms (ECGs), and clinical laboratory tests (hematology, serum safety chemistry, and urinalysis). Tests for growth and development (eg, estradiol, testosterone, dehydroepiandrosterone, follicle stimulating hormone, luteinizing hormone, adrenocorticotropic hormone, and cortisol) will be assessed at the Week 48, 96 and 156 visits. Samples will be collected for immunogenicity testing. Injection site reactions will be assessed at each dosing visit.