Lerodalcibep for High Cholesterol

(LIBerate Kids Trial)

Not yet recruiting at 2 trial locations

Overseen By Kate Caldwell Sr. Director, Clinical Development, LIB Therapeutics, LLC, BS MT(ASCP)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: LIB Therapeutics LLC

Must be taking: Statins, Ezetimibe

Must not be taking: Mipomersen, Lomitapide, Gemfibrozil

Disqualifiers: HoFH, Liver disease, Uncontrolled diabetes, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 20

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Lerochol (also known as Lerodalcibep) to determine its effectiveness in reducing LDL cholesterol, often referred to as "bad" cholesterol, in children and teens with inherited high cholesterol. Researchers aim to assess whether Lerochol effectively lowers cholesterol levels and is safe for use. Participants with long-term high cholesterol, who maintain a stable diet, and take cholesterol-lowering medications like statins might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to the development of a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires participants to be on a stable diet and lipid-lowering oral therapies like statins for at least 6 weeks before joining. If you are taking a PCSK9 inhibitor, you must stop it for a specific period before starting the trial: more than 8 weeks for PCSK9 mAb and 360 days for siRNA PCSK9 inhibitors.

Is there any evidence suggesting that lerodalcibep is likely to be safe for humans?

Research has shown that lerodalcibep is generally safe and effective. In studies, participants taking lerodalcibep experienced a significant reduction in LDL cholesterol levels without major safety issues. Long-term results indicate it remains effective and safe even after a year of use. No reports of serious side effects suggest it is safe for individuals with inherited high cholesterol. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Lerodalcibep is unique because it targets PCSK9, a protein that plays a crucial role in cholesterol regulation. Unlike traditional statins, which primarily decrease cholesterol production in the liver, lerodalcibep works by preventing PCSK9 from breaking down LDL receptors, thus enhancing the body's ability to clear LDL cholesterol from the blood. This monthly subcutaneous injection offers a new approach for patients who may not respond adequately to or tolerate current therapies. Researchers are excited about lerodalcibep because it could provide an effective alternative for managing high cholesterol, especially for those struggling with existing medications.

What evidence suggests that lerodalcibep might be an effective treatment for high cholesterol?

In this trial, participants will receive either lerodalcibep or a placebo. Previous studies have shown that lerodalcibep effectively lowers LDL cholesterol, the "bad" cholesterol linked to heart disease. Research indicates that over 90% of patients taking lerodalcibep experienced at least a 50% reduction in LDL levels. Specifically, one study found that 86.2% of patients achieved this reduction, and 69.6% reached their cholesterol goals. This treatment blocks a protein called PCSK9, enabling the body to remove more LDL cholesterol from the blood. These findings suggest lerodalcibep could be a powerful option for managing inherited high cholesterol. ^{1 2 3 4 6}

Who Is on the Research Team?

David Kallend, MB BS

Principal Investigator

Chief Medical Officer, LIB Therapeutics, LLC

Are You a Good Fit for This Trial?

This trial is for children aged 6 to 17 with inherited high cholesterol (HeFH) who are on a stable diet and taking the highest dose of oral cholesterol-lowering drugs they can tolerate. They must be able to visit the clinic monthly, fast overnight before visits, and undergo various health checks.

Inclusion Criteria

I have been diagnosed with a form of high cholesterol that could be genetic.

Provision of written and signed informed consent/assent prior to any study-specific procedure

I am between 6 and 17 years old.

Exclusion Criteria

Patients who cannot be available for Protocol-required study visits or procedures

I have severe heart failure or poor heart pump function.

My high blood pressure is not under control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 11 weeks

Treatment

Participants receive monthly subcutaneous injections of lerodalcibep or placebo for 24 weeks

24 weeks

Monthly visits for 20 weeks, then every 2 weeks for Weeks 22 and 24

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Lerodalcibep

Trial Overview The study tests if Lerodalcibep (Lerochol), given as a monthly shot, lowers 'bad' LDL cholesterol in kids with HeFH more effectively than a placebo. It's double-blind, meaning neither doctors nor patients know who gets Lerochol or the dummy injection.

How Is the Trial Designed?

Group I: Arm 1: lerodalcibep 300 mg Active Control 1 Intervention

monthly Subcutaneous injection

Group II: Arm 2: Placebo Placebo Group 1 Intervention

monthly subcutaneous injection

Who Is Running the Clinical Trial?

LIB Therapeutics LLC

Lead Sponsor

Trials

11

Recruited

4,900+

Citations

1. jamanetwork.com jamanetwork.com/journals/jamacardiology/fullarticle/2820248

Efficacy and Safety of Lerodalcibep in Patients With or at ... Efficacy and safety of more intensive lowering of LDL cholesterol: a meta-analysis of data from 170,000 participants in 26 randomised trials.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10590131/

Long-term efficacy and safety of lerodalcibep in heterozygous ... A reduction in LDL-C of ≥50% was achieved in 275 subjects (86.2%) with lerodalcibep, and ESC LDL-C targets were achieved in 222 (69.6%) subjects (Figure 3).

3. acc.org acc.org/Latest-in-Cardiology/Articles/2024/04/02/17/02/sun-8am-liberate-hr-acc-2024

LIBerate-HR: Does Novel PCSK9 Inhibitor Lerodalcibep ... More than 90% of patients in the lerodalcibep group achieved a reduction of ≥50% in LDL-C and attained the target LDL-C level for their risk ...

4. atherosclerosis-journal.com atherosclerosis-journal.com/article/S0021-9150(25)00515-5/fulltext

Efficacy of lerodalcibep, a third generation PCSK9 inhibitor ... Conclusions: Lerodalcibep 300 mg QM significantly and consistently reduced LDL-C in HeFH subjects with identified FH causing variants and in those without known ...

5. jacc.org jacc.org/doi/10.1016/S0735-1097%2824%2903694-5

LONG-TERM EFFICACY AND SAFETY OF ... Lerodalcibep, a novel small binding protein inhibitor of circulating PCSK9, reduced placebo-adjusted LDL-cholesterol (LDL-C) by a mean(SE) of 58.61(3.25)% ...

6. atherosclerosis-journal.com atherosclerosis-journal.com/article/S0021-9150(24)00973-0/fulltext

Randomized, open-label, study comparing efficacy and ... Randomized, open-label, study comparing efficacy and safety of lerodalcibep to inclisiran in patients with CVD or at high risk for CVD requiring additional LDL- ...