Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Homozygous Familial Hypercholesterolemia Clinical Trial

Official title:

Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04034485
• Other study ID #: LIB003-003
• Status: Completed
• Phase: Phase 3
• Start date: December 7, 2019
• Est. completion date: January 30, 2023

Study information

• Verified date: March 2023
• Source: LIB Therapeutics LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Description:

Patients with verified HoFH on stable and continuing doses of oral lipid lowering therapy will be randomized to either evolocumab 420 mg Q4W or LIB003 300 mg Q4W for 24 weeks (Period A). At Week 24, subjects will be crossed over to LIB003 if they were on evolocumab and vice versa for the next 24 weeks (Period B).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 65
• Est. completion date: January 30, 2023
• Est. primary completion date: September 12, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

• prior or active clinical condition or acute and/or unstable systemic disease compromising subject inclusion

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• lerodalcibep
PCSK9 inhibitor
• evolocumab
PCSK9 inhibitor

Locations

Country	Name	City	State
India	CIMS Hospital Pvt. Ltd	Ahmedabad
India	VMMC & Safdarjung Hospital	Delhi	NCT
India	G.B. Pant Institute of Postgraduate Medical Education & Research	New Delhi
Israel	Department of Medicine, Hadassah University Hospital	Jerusalem
Israel	Rabin Medical Center, Beilinson Hospital,	Petah Tikva
Norway	Lipid Clinic, Oslo University Hospital	Oslo
South Africa	Division of Lipidology, Department of Medicine University of Cape Town	Cape Town	Western Province
South Africa	Carbohydrate and Lipid Metabolism Research Unit	Johannesburg	Gauteng
Turkey	Afyonkarahisar Health Sciences University	Afyon
Turkey	Ege University Medical School	Izmir	Bornova
United States	Metabolic & Atherosclerosis Research Center (MARC)	Cincinnati	Ohio
United States	NorthShore University Health System	Evanston	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
LIB Therapeutics LLC

Countries where clinical trial is conducted

United States,  India,  Israel,  Norway,  South Africa,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Percent reduction in lipoprotein (a) [Lp(a)] at week 24	Change in serum Lp(a) from baseline after 24 weeks	baseline to 24 weeks on each treatment
Other	Percent reduction in apolipoprotein B (Apo B) at week 24	Change in serum Apo B from baseline after 24 weeks	baseline to 24 weeks on each treatment
Other	Presence of anti LIB003 antibodies (ADAs)	Measurement of ADAs at baseline and various intervals	baseline to 24 weeks
Primary	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	The incidence and severity of treatment emergent adverse events (TEAEs)	safety and tolerability will be based on the incidence and severity of treatment emergent adverse events	baseline to 24 weeks on each treatment