Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety … (NCT07102511) | Clinical Trial Compass
Not Yet RecruitingPhase 3
Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH
United States, South Africa, Turkey (Türkiye)150 participantsStarted 2025-09-01
Plain-language 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.
Who can participate
Age range6 Years – 17 Years
SexALL
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Inclusion criteria
✓. 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;
✓. 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);
✓. 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;
✓. Females of childbearing potential must be using a highly effective form of contraception if sexually active and have negative urine pregnancy test at the last Screening Visit; Note: Highly effective methods of birth control include refraining from heterosexual sexual intercourse during the entire period of risk, birth control pills or patches, intrauterine devices (IUDs), sexual activity with a male partner who has had a vasectomy, condom or diaphragm or cervical cap with spermicide or IUD, oral, implantable, or injectable contraceptives.
✓. Male patients will either be surgically sterile or agree to use the following forms of contraception: 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; and
Exclusion criteria
✕. 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;
✕. Females of childbearing potential who are sexually active, not using or unwilling to use a highly effective form of contraception, pregnant or breastfeeding, or who have a positive urine pregnancy test at the last Screening Visit; Note: Highly effective methods of birth control include refraining from heterosexual sexual intercourse during the entire period of risk, birth control pills or patches, IUDs, sexual activity with a male partner who has had a vasectomy, condom or diaphragm or cervical cap with spermicide or IUD, oral, implantable, or injectable contraceptives.
✕. Moderate to severe renal dysfunction, defined as an estimated glomerular filtration rate \<30 mL/min/1.73m2 at the Screening Visit;
✕. 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);
✕. 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;