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

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.

Inclusion criteria

• Provision of written and signed informed consent/assent of the LIB003-016 trial prior to any study-specific procedure;
• Completion of the LIB003-008 study having received the last dose of study drug at Week 20 and completed the Week 24 visit;
• Male or female, 6 to 17 years of age (defined as from 6 to less than 18 years of age), at the first Screening Visit of the LIB003-008 study;
• Weight of >18 kg (40 lbs) and BMI <17 and >42 kg/m2;
• On stable diet and lipid-lowering oral drug therapy (statins, ezetimibe, bile-acid sequestrants) or combinations thereof (excluded oral lipid-lowering agents are include mipomersen, lomitapide, and gemfibrozil);

Exclusion criteria

• History of any prior or active clinical condition or acute and/or unstable systemic disease compromising patient inclusion, at the discretion of the Investigator, which in the Investigator's opinion, would not be suitable for the study from a patient safety consideration or could interfere with the results of the study;
• Females of childbearing potential who are sexually active, not using or unwilling to use a highly effective form of contraception during the study and until 60 days after last dose of study drug, pregnant or breastfeeding, or who have a positive urine pregnancy test at the last Screening Visit;
• Patients who cannot be available for Protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge;
• A history, during the LIB003-008 study of prescription drug abuse, illicit drug use, or alcohol abuse according to medical history;
• Have any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study;