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.
Provision of written and signed informed consent/assent prior to any study-specific procedure;
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;
Weight of more than 18 kg (40 lbs) and BMI more than 17 and less than42 kg/m2;
Diagnosis of definite, probable, or possible HeFH based on clinical criteria (SB Register criteria or genotyping and at the defined eligibility visit (Screening Visit or post washout/stabilization); a calculated LDL-C (Friedewald) equal or above 130 mg/dL or, if the patient has additional risk factors as defined , a calculated LDL-C (Friedewald) equal or above100 mg/dL; and TG below 400 mg/dL while on stable lipid-lowering oral drug therapy (eg, maximally tolerated statin with or without ezetimibe); Note: Patients unable to tolerate approved doses of a statin may take lower than approved doses and less frequently than daily as long as the dose and dosing frequency is consistent per the Investigator's judgment. Patients with documented intolerance to statins may also participate;
Use of prohibited oral lipid-lowering agents mipomersen or lomitapide within 6 months of screening or gemfibrozil within 6 weeks of the Screening Visit;
LDL or plasma apheresis within 2 months prior to Day 1;
Documented history of HoFH defined as clinical and/or genetic with true HoFH (ie, identical pathogenic variants), compound heterozygous (ie, 2 different pathogenic LDLR variants) or combined heterozygous (2 different pathogenic FH variants such as LDLR plus ApoB or LDLR plus PCSK9 gain-of function);
History of any prior or active clinical condition or acute and/or unstable systemic disease compromising patient inclusion, at the discretion of the Investigator, including but not limited to clinically significant pulmonary, hematologic, gastrointestinal, endocrine (excluding diabetes), immunologic, dermatologic, neurologic, or psychiatric disease, 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;