NCT04034485

Brief Summary

To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks \[Q4W\]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH on stable diet and oral LDL-C-lowering drug therapy

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
65

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Dec 2019

Typical duration for phase_3

Geographic Reach
6 countries

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 22, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 26, 2019

Completed
4 months until next milestone

Study Start

First participant enrolled

December 7, 2019

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 12, 2022

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 30, 2023

Completed
Last Updated

March 29, 2023

Status Verified

March 1, 2023

Enrollment Period

2.8 years

First QC Date

July 22, 2019

Last Update Submit

March 27, 2023

Conditions

Homozygous Familial Hypercholesterolemia

Keywords

LDL cholesterolPCSK9 inhibition

Outcome Measures

Primary Outcomes (1)

  • Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24

    Change in serum LDL-C from baseline after 24 weeks

    baseline to 24 weeks on each treatment

Secondary Outcomes (1)

  • The incidence and severity of treatment emergent adverse events (TEAEs)

    baseline to 24 weeks on each treatment

Other Outcomes (3)

  • Percent reduction in lipoprotein (a) [Lp(a)] at week 24

    baseline to 24 weeks on each treatment

  • Percent reduction in apolipoprotein B (Apo B) at week 24

    baseline to 24 weeks on each treatment

  • Presence of anti LIB003 antibodies (ADAs)

    baseline to 24 weeks

Study Arms (2)

LIB003 (lerodalcibep)

EXPERIMENTAL

300 mg SC Q4W

Drug: lerodalcibep

evolocumab

ACTIVE COMPARATOR

420 mg SC Q4W

Drug: evolocumab

Interventions

lerodalcibepDRUG

PCSK9 inhibitor

Also known as: LIB003
LIB003 (lerodalcibep)
evolocumabDRUG

PCSK9 inhibitor

Also known as: Repatha
evolocumab

Eligibility Criteria

Age10 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • HoFH diagnosed clinically and confirmed by genotyping
  • Weight of \>30 kg and body mass index (BMI) \>17 and \<40 kg/m2
  • stable diet and lipid-lowering oral therapies for at least 4 weeks

You may not qualify if:

  • mipomersen within 6 months of screening;
  • LDL or plasma apheresis \<2 months prior to randomization
  • history of non-response to PCSK9 mAb or presence of receptor negative/null LDLR activity expected to result in non-response to PCSK9 inhibition

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

NorthShore University Health System

Evanston, Illinois, 60201, United States

Location

Metabolic & Atherosclerosis Research Center (MARC)

Cincinnati, Ohio, 45227, United States

Location

VMMC & Safdarjung Hospital

Delhi, National Capital Territory of Delhi, India

Location

CIMS Hospital Pvt. Ltd

Ahmedabad, India

Location

G.B. Pant Institute of Postgraduate Medical Education & Research

New Delhi, 110002, India

Location

Department of Medicine, Hadassah University Hospital

Jerusalem, 12000, Israel

Location

Rabin Medical Center, Beilinson Hospital,

Petah Tikva, 49100, Israel

Location

Lipid Clinic, Oslo University Hospital

Oslo, 0586, Norway

Location

Carbohydrate and Lipid Metabolism Research Unit

Johannesburg, Gauteng, 2193, South Africa

Location

Division of Lipidology, Department of Medicine University of Cape Town

Cape Town, Western Province, 7925, South Africa

Location

Ege University Medical School

Izmir, Bornova, 35040, Turkey (Türkiye)

Location

Afyonkarahisar Health Sciences University

Afyonkarahisar, Turkey (Türkiye)

Location

Related Publications (1)

  • Raal FJ, Mehta V, Kayikcioglu M, Blom D, Gupta P, Elis A, Turner T, Daniels C, Vest J, Mitchell T, Caldwell K, Bahassi EM, Kallend D, Stein EA. Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, non-inferiority trial. Lancet Diabetes Endocrinol. 2025 Mar;13(3):178-187. doi: 10.1016/S2213-8587(24)00313-9. Epub 2025 Jan 24.

    PMID: 39870096DERIVED

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

evolocumab

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Evan A Stein, MD PhD

    LIB Therapeutics

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Masking Details
treatment is open label but lipid results are masked to participant, investigator and sponsor
Purpose
TREATMENT
Intervention Model
CROSSOVER
Model Details: open label, randomized, cross-over
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 22, 2019

First Posted

July 26, 2019

Study Start

December 7, 2019

Primary Completion

September 12, 2022

Study Completion

January 30, 2023

Last Updated

March 29, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

IL(2)IN(3)US(2)NO(1)ZA(2)TU(2)