Randomized, Placebo-Controlled, Double-Blind, Phase 3b 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
LIB Therapeutics LLC
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.
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…
Eligibility
- Age range
- 6–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Provision of written and signed informed consent/assent prior to any study-specific procedure; 2. 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; 3. Weight of more than 18 kg (40 lbs) and BMI more than 17 and less than42 kg/m2; 4. 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 h…
Interventions
- Biologicallerodalcibep 300 mg Monthly SC
lerodalcibep or placebo
Locations (3)
- Metabolic & Atherosclerosis Research CenterCincinnati, Ohio
- Lipid Clinic Wits UniversityJohannesburg, Gauteng
- Ege Universitesi Tip FakultesiIzmir