Authorised Therapeutic confirmatory (Phase III) Heterozygous Familial Hypercholesterolemia

Study to Evaluate the Efficacy and Safety of Lerodalcibep for the Reduction of Cholesterol in Children and Adolescents with Heterozygous Familial Hypercholesterolemia

EU CTIS ID: 2025-524214-28-00

What this study is testing

To assess the LDL-C reductions at Week 24 with monthly dosing of lerodalcibep 300 mg compared to placebo, in pediatric patients 6 to 17 years of age, with Heterozygous Familial Hypercholesterolemia on a stable diet and maximally tolerated oral LDL-C lowering drug therapy

Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Provision of written and signed informed consent/assent prior to any study-specific procedure
10. Male patients must refrain from sperm donation until 90 days following the last dose of study drug
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 than 42 kg/m2
4. Diagnosis of definite or probable Heterozygous Familial Hypercholesterolemia (HeFH) based on clinical criteria (SB Register, MEDPED or DLCN 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 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.
5. On a stable diet and lipid-lowering oral therapies (statins, ezetimibe, bile-acid sequestrants) or combinations thereof for at least 6 weeks (excluded oral lipid-lowering agents include mipomersen, lomitapide, and gemfibrozil)

You likely can't join if

1. Use of prohibited oral lipid-lowering agents mipomersen or lomitapide within 6 months of screening or gemfibrozil within 6 weeks of the Screening Visit
10. Uncontrolled serious cardiac arrhythmia (sustained ventricular tachycardia, frequent non sustained ventricular tachycardia, any ventricular fibrillation episode, wide-complex tachycardia, atrial fibrillation with rapid ventricular response, and severe second-degree or third degree atrioventricular block), myocardial infarction (MI), unstable angina, percutaneous coronary intervention, coronary artery bypass grafting, placement of implantable cardioverter defibrillator or biventricular pacemaker, aortic valve surgery, or stroke within 3 months prior to enrollment (the day patient signs the informed consent/assent and first procedure is performed)
11. Planned cardiac surgery or revascularization
12. New York Heart Association III-IV heart failure; or patients with last documented left ventricular ejection fraction <30% by standard of care assessments (eg, echocardiography, cardiac magnetic resonance imaging, nuclear imaging, computed tomography angiography, or angiography with ventriculogram), within 12 months
13. Uncontrolled hypertension defined as on treatment diastolic or systolic BP ≥95th percentile for age and sex
14. Enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives since ending another investigational device or drug study(ies), or receiving other investigational agent(s); such as PCSK9 or ANGPTL3 or Lp(a) siRNA or locked nucleic acid reducing agents within 12 months of the Screening Visit

See the full eligibility criteria

Who can join

1. Provision of written and signed informed consent/assent prior to any study-specific procedure
10. Male patients must refrain from sperm donation until 90 days following the last dose of study drug
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 than 42 kg/m2
4. Diagnosis of definite or probable Heterozygous Familial Hypercholesterolemia (HeFH) based on clinical criteria (SB Register, MEDPED or DLCN 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 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.
5. On a stable diet and lipid-lowering oral therapies (statins, ezetimibe, bile-acid sequestrants) or combinations thereof for at least 6 weeks (excluded oral lipid-lowering agents include mipomersen, lomitapide, and gemfibrozil)
6. Patients on a PCSK9 mAb must undergo a washout period of ≥8 weeks after the last dose. For patients who have received an siRNA PCSK9 inhibitor the washout period is 360 days post last dose
7. Females of childbearing potential must be using a highly effective form of contraception as specified in the study protocol during the study and until 60 days after last dose of study drug if sexually active and have negative urine pregnancy test during the trial and at the last Screening Visit
8. Females of child bearing potential are not permitted to donate oocytes during exposure to the IMP or for 90 days after the last dose of study drug
9. Male patients will either be surgically sterile or agree to use the following forms of contraception until 90 days after the last dose of study drug: male or female condom with spermicide and a female partner who is sterile or who agrees to use the following contraceptives: diaphragm or cervical cap with spermicide; or IUD, oral, implantable, or injectable contraceptives

What rules you out

1. Use of prohibited oral lipid-lowering agents mipomersen or lomitapide within 6 months of screening or gemfibrozil within 6 weeks of the Screening Visit
10. Uncontrolled serious cardiac arrhythmia (sustained ventricular tachycardia, frequent non sustained ventricular tachycardia, any ventricular fibrillation episode, wide-complex tachycardia, atrial fibrillation with rapid ventricular response, and severe second-degree or third degree atrioventricular block), myocardial infarction (MI), unstable angina, percutaneous coronary intervention, coronary artery bypass grafting, placement of implantable cardioverter defibrillator or biventricular pacemaker, aortic valve surgery, or stroke within 3 months prior to enrollment (the day patient signs the informed consent/assent and first procedure is performed)
11. Planned cardiac surgery or revascularization
12. New York Heart Association III-IV heart failure; or patients with last documented left ventricular ejection fraction <30% by standard of care assessments (eg, echocardiography, cardiac magnetic resonance imaging, nuclear imaging, computed tomography angiography, or angiography with ventriculogram), within 12 months
13. Uncontrolled hypertension defined as on treatment diastolic or systolic BP ≥95th percentile for age and sex
14. Enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives since ending another investigational device or drug study(ies), or receiving other investigational agent(s); such as PCSK9 or ANGPTL3 or Lp(a) siRNA or locked nucleic acid reducing agents within 12 months of the Screening Visit
15. Unexplained CK >5 × ULN, unless related to exercise or unusual activity in which case 1 repeat test is allowed
16. Patients who cannot be available for Protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge
17. A history, within 6 months prior to screening, of prescription drug abuse, illicit drug use, or alcohol abuse according to medical history
18. Donated or lost a significant volume (>500 mL) of blood or plasma within 30 days prior to Day 1
19. Had a blood transfusion within 4 weeks of randomization or known diagnosis of human immunodeficiency virus
2. LDL or plasma apheresis within 2 months prior to Day 1
20. Previous treatment with lerodalcibep or any adnectin product
21. Have any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study
22. An employee or family member of the Investigator or study site personnel
3. Documented history of Homozygous Familial Hypercholesterolemia (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)
4. 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
5. Females of childbearing potential who are sexually active, not using or unwilling to use a highly effective form of contraception as specified in the study protocol 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
6. Moderate to severe renal dysfunction, defined as an estimated glomerular filtration rate <30 mL/min/1.73m2 at the Screening Visit
7. Active liver disease or hepatic dysfunction (eg, cirrhosis, alcoholic liver disease, known hepatitis B or hepatitis C, autoimmune hepatitis, liver failure, liver cancer), history of liver transplant, and/or AST or ALT >2.5 × the ULN based on age as determined by central laboratory analysis at screening (tests that result in ALT or AST up to 3 × ULN may have 1 repeat test to confirm eligibility during the Screening Period)
8. Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) below the lower limit of normal (LLN) or >1.5 × ULN, respectively, at the Screening Visit. If TSH is above/below these cut-off points, the patient can enter if the free triiodothyronine (FT3) is within the reference range. If controlled, then treatment should be stable for at least 3 months prior to the Screening Visit
9. Uncontrolled Type 1 or Type 2 diabetes mellitus (defined as fasting glucose above 200 mg/dL and HbA1c of above 9%)

The study team makes the final eligibility decision.

Where it's taking place

New Zealand
Turkey
Israel
United States
India
South Africa

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include New Zealand; Turkey; Israel; United States; India; South Africa. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.