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Study to Assess the Efficacy and Safety of LIB003 in HeFH Patients on Oral Lipid Therapy Needing Further LDL-C Reduction (LIBerate-FH)

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ClinicalTrials.gov Identifier: NCT04797104
Recruitment Status : Active, not recruiting
First Posted : March 15, 2021
Last Update Posted : November 4, 2022
Medpace, Inc.
Information provided by (Responsible Party):
LIB Therapeutics LLC

Brief Summary:
This study is to assess LDL-C reductions at Week 24 and the mean of Weeks 22 and 24 with monthly Q4W (≤31 days) dosing of LIB003 300 mg administered subcutaneously (SC) compared to placebo in patients 18 years or older with Heterozygous FH on stable diet and oral LDL-C lowering drug therapy.

Condition or disease Intervention/treatment Phase
Heterozygous Familial Hypercholesterolemia Drug: lerodalcibep Phase 3

Detailed Description:
A randomized, double-blind, placebo-controlled, Phase 3 study of 24 weeks duration. Approximately 600 males and females aged ≥18 years with clinical or genetic heterozygous FH who fulfill the inclusion and exclusion criteria will be enrolled. Patients will be randomized in a 2:1 ratio to LIB003 (400 patients) or placebo (200 patients) administered SC Q4W (≤31 days). The study will consist of a Screening Period and a Treatment Period. The total study duration will be up to 35 weeks which includes up to an 11-week Screening Period (which may include up to a 8-week washout) and 24 weeks of study drug treatment.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 450 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized double blind placebo controlled
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: double blind to treatment (LIB003 or placebo) and lipid data
Primary Purpose: Treatment
Official Title: Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Heterozygous FH Patients on Stable Lipid-Lowering Therapy Requiring Additional LDL-C Reduction
Actual Study Start Date : April 22, 2021
Estimated Primary Completion Date : April 30, 2023
Estimated Study Completion Date : May 31, 2023

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: LIB003 (lerodalcibep)
300 mg (1.2 mL) SC Q4W
Drug: lerodalcibep
300 mg Q4W
Other Name: LIB003

Placebo Comparator: Placebo
1.2 mL SC Q4W
Drug: lerodalcibep
300 mg Q4W
Other Name: LIB003

Primary Outcome Measures :
  1. Change from baseline compared to placebo in LDL-C level [ Time Frame: 24 weeks ]
    LS mean percent change in LDL-cholesterol compared to placebo

Secondary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events as assessed by Medical Dictionary for Regulatory Activities [ Time Frame: 24 weeks ]
    Incidence of Treatment-Emergent Adverse Events (TEAE) as assessed by Medical Dictionary for Regulatory Activities as mild, moderate or severe compared to placebo

  2. Change in serum free PCSK9 with LIB003 compared to placebo [ Time Frame: 24 weeks ]
    Change in percent LS mean between LIB003 and placebo

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Provision of written and signed informed consent prior to any study-specific procedure;
  • Weight of ≥40 kg (88 lbs) and body mass index (BMI) ≥17 and ≤42 kg/m2;
  • Diagnosis of definite, probable or possible HeFH based either on clinical criteria (Simon Broome register criteria or Dutch Lipid Clinics [DLC] Network Criteria) or genotyping and at the defined eligibility visit (screening or post washout/stabilization)
  • LDL-C ≥70 mg/dL (if very-high risk for CVD) or ≥100 mg/dL (if high risk for CVD) and TG ≤400 mg/dL while on stable lipid lowering oral drug therapy (eg, maximally tolerated statin with or without ezetimibe); 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.
  • Patients with documentation of inability to tolerate any statin at any dose, or history of rhabdomyolysis, and unable to tolerate any other allowed oral lipid lowering agent, and thus on no lipid lowering therapy must have an LDL-C ≥190 mg/dL (4.9 mmol/L) at the Screening Visit unless they have a documented pathogenic FH variant;
  • Stable diet and other lipid lowering oral therapies besides statins and ezetimibe including bile-acid sequestrants, OM-3 compounds, fenofibrate, bezafibrate, nicotinic acid and bempedoic acid or combinations thereof for at least 4 weeks
  • Patients on a PCSK9 mAb at a dose of 75 mg, 140 mg, or 150 mg Q2W must undergo a washout period of ≥4 weeks after the last dose; for those on a dose of 300 mg or 420 mg Q4W (≤31 days) the washout period is ≥8 weeks following 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

Exclusion Criteria:

  • Use of prohibited oral lipid lowering agents mipomersen or lomitapide within 6 months of screening, gemfibrozil within 6 weeks of the Screening Visit or LDL/plasma apheresis within 2 months prior to Day 1;
  • Documented history of HoFH defined clinically or genetically
  • History of any prior or active clinical condition or acute and/or unstable systemic disease compromising patient inclusion, at the discretion of the Investigator.
  • 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;
  • Moderate to severe renal dysfunction, defined as an eGFR <30 mL/min/1.73m2
  • Active liver disease or hepatic dysfunction, history of liver transplant, and/or AST or ALT >2.5 × the ULN
  • Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by TSH <LLN or >1.5 × ULN, respectively,
  • Uncontrolled Type 1 or Type 2 DM (fasting glucose≥200 mg/dL or HbA1c of ≥9%;
  • Planned cardiac surgery or revascularization;
  • New York Heart Association III-IV heart failure
  • Previous treatment with LIB003 or any adnectin product;
  • Any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04797104

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United States, Ohio
Metabolic & Atherosclerosis Research Center (MARC)
Cincinnati, Ohio, United States, 45227
Department of Medicine, Hadassah University Hospital
Jerusalem, Israel, 12000
Rabin Medical Center, Beilinson Hospital,
Petah Tikva, Israel, 49100
Lipid Clinic, Oslo University Hospital
Oslo, Norway, 0586
South Africa
Carbohydrate and Lipid Metabolism Research Unit
Johannesburg, Gauteng, South Africa, 2193
Division of Lipidology, Department of Medicine University of Cape Town
Cape Town, Western Province, South Africa, 7925
Ege University Medical School
İzmir, Bornova, Turkey, 35040
Sponsors and Collaborators
LIB Therapeutics LLC
Medpace, Inc.
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Study Director: David Kallend, MB BCh LIB Therapeutics
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Responsible Party: LIB Therapeutics LLC
ClinicalTrials.gov Identifier: NCT04797104    
Other Study ID Numbers: LIB003-004
First Posted: March 15, 2021    Key Record Dates
Last Update Posted: November 4, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by LIB Therapeutics LLC:
PCSK9 inhibitor
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn