Heterozygous Familial Hypercholesterolemia (HeFH)Phase 3NCT07775339
Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents With Familial Hypercholesterolemia
A Phase 3 study testing Lerodalcibep in people with Heterozygous Familial Hypercholesterolemia (HeFH), run by LIB Therapeutics LLC at 5 sites in 5 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia (HeFH)
- What is being tested?
- Lerodalcibep
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 20 Jan 2027to 31 Dec 2031
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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);
Who cannot join
- 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;
Trial locations and contacts
5 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 5 countries
India1 site
G.B. Pant Institute of Postgraduate Medical Education & Research
Delhi, 110002
About this study
Official title: 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:
Read more
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.
Read the detailed description
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.
- Being tested
- Lerodalcibep
- Keywords
- PCSK9 inhibitor, LDL-C, HeFH, lerodalcibep, pediatric
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LerodalcibepDrug
300 mg monthly
| Study group | Role | What they receive |
|---|---|---|
| Lerodalcibep | Experimental | lerodalcibep 300 mg monthly Receives: Drug: Lerodalcibep |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in LDL cholesterol at Week 156 from baseline
The primary objective of this study is to assess the change in LDL-C (by Friedewald Formula) at Week 156 with monthly lerodalcibep 300 mg QM
Measured: 156 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2026
- Study start20 Jan 2027 (estimated)
- Primary completion31 Jul 2031 (estimated)
- Study completion31 Dec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous Familial Hypercholesterolemia (HeFH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- openlabel extension trial with single biologi drug - lerodalcibep
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- LIB Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07775339
- Sponsor's protocol ID
- LIB003-016
- Other IDs
- 2026-528022-38-00
- Status last verified
- Aug 2026
- First submitted
- 16 Aug 2026
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided