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

Open Label Phase 3b Study to Evaluate the Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia (LIBerate-Kids OLE)

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.

Inclusion Criteria:

1. Provision of written and signed informed consent/assent of the LIB003-016 trial prior to any study-specific procedure;
2. Completion of the LIB003-008 study having received the last dose of study drug at Week 20 and completed the Week 24 visit;
3. 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;
4. Weight of >18 kg (40 lbs) and BMI <17 and >42 kg/m2;
5. 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:

1. 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;
2. 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;
3. Patients who cannot be available for Protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge;
4. A history, during the LIB003-008 study of prescription drug abuse, illicit drug use, or alcohol abuse according to medical history;
5. Have any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study;