Back to resultsPhase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
Primary Purpose
Homozygous Familial Hypercholesterolemia
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
lerodalcibep
evolocumab
Sponsored by
About this trial
This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia focused on measuring LDL cholesterol, PCSK9 inhibition
Eligibility Criteria
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
Sites / Locations
- NorthShore University Health System
- Metabolic & Atherosclerosis Research Center (MARC)
- VMMC & Safdarjung Hospital
- CIMS Hospital Pvt. Ltd
- G.B. Pant Institute of Postgraduate Medical Education & Research
- Department of Medicine, Hadassah University Hospital
- Rabin Medical Center, Beilinson Hospital,
- Lipid Clinic, Oslo University Hospital
- Carbohydrate and Lipid Metabolism Research Unit
- Division of Lipidology, Department of Medicine University of Cape Town
- Ege University Medical School
- Afyonkarahisar Health Sciences University
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
LIB003 (lerodalcibep)
evolocumab
Arm Description
300 mg SC Q4W
420 mg SC Q4W
Outcomes
Primary Outcome Measures
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
Change in serum LDL-C from baseline after 24 weeks
Secondary Outcome Measures
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04034485
Brief Title
Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
December 7, 2019 (Actual)
Primary Completion Date
September 12, 2022 (Actual)
Study Completion Date
January 30, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
LIB Therapeutics LLC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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
Detailed 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Homozygous Familial Hypercholesterolemia
Keywords
LDL cholesterol, PCSK9 inhibition
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Model Description
open label, randomized, cross-over
Masking
None (Open Label)
Masking Description
treatment is open label but lipid results are masked to participant, investigator and sponsor
Allocation
Randomized
Enrollment
65 (Actual)
8. Arms, Groups, and Interventions
Arm Title
LIB003 (lerodalcibep)
Arm Type
Experimental
Arm Description
300 mg SC Q4W
Arm Title
evolocumab
Arm Type
Active Comparator
Arm Description
420 mg SC Q4W
Intervention Type
Drug
Intervention Name(s)
lerodalcibep
Other Intervention Name(s)
LIB003
Intervention Description
PCSK9 inhibitor
Intervention Type
Drug
Intervention Name(s)
evolocumab
Other Intervention Name(s)
Repatha
Intervention Description
PCSK9 inhibitor
Primary Outcome Measure Information:
Title
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
Description
Change in serum LDL-C from baseline after 24 weeks
Time Frame
baseline to 24 weeks on each treatment
Secondary Outcome Measure Information:
Title
The incidence and severity of treatment emergent adverse events (TEAEs)
Description
safety and tolerability will be based on the incidence and severity of treatment emergent adverse events
Time Frame
baseline to 24 weeks on each treatment
Other Pre-specified Outcome Measures:
Title
Percent reduction in lipoprotein (a) [Lp(a)] at week 24
Description
Change in serum Lp(a) from baseline after 24 weeks
Time Frame
baseline to 24 weeks on each treatment
Title
Percent reduction in apolipoprotein B (Apo B) at week 24
Description
Change in serum Apo B from baseline after 24 weeks
Time Frame
baseline to 24 weeks on each treatment
Title
Presence of anti LIB003 antibodies (ADAs)
Description
Measurement of ADAs at baseline and various intervals
Time Frame
baseline to 24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Evan A Stein, MD PhD
Organizational Affiliation
LIB Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
NorthShore University Health System
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60201
Country
United States
Facility Name
Metabolic & Atherosclerosis Research Center (MARC)
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45227
Country
United States
Facility Name
VMMC & Safdarjung Hospital
City
Delhi
State/Province
NCT
Country
India
Facility Name
CIMS Hospital Pvt. Ltd
City
Ahmedabad
Country
India
Facility Name
G.B. Pant Institute of Postgraduate Medical Education & Research
City
New Delhi
ZIP/Postal Code
110002
Country
India
Facility Name
Department of Medicine, Hadassah University Hospital
City
Jerusalem
ZIP/Postal Code
12000
Country
Israel
Facility Name
Rabin Medical Center, Beilinson Hospital,
City
Petah Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Lipid Clinic, Oslo University Hospital
City
Oslo
ZIP/Postal Code
0586
Country
Norway
Facility Name
Carbohydrate and Lipid Metabolism Research Unit
City
Johannesburg
State/Province
Gauteng
ZIP/Postal Code
2193
Country
South Africa
Facility Name
Division of Lipidology, Department of Medicine University of Cape Town
City
Cape Town
State/Province
Western Province
ZIP/Postal Code
7925
Country
South Africa
Facility Name
Ege University Medical School
City
İzmir
State/Province
Bornova
ZIP/Postal Code
35040
Country
Turkey
Facility Name
Afyonkarahisar Health Sciences University
City
Afyon
Country
Turkey
12. IPD Sharing Statement
Plan to Share IPD
No
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Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
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