Homozygous Familial HypercholesterolemiaPhase 3NCT04034485
Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH
A Phase 3 study testing lerodalcibep and evolocumab in people with Homozygous Familial Hypercholesterolemia, run by LIB Therapeutics LLC at 12 sites in 6 countries.
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 Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- lerodalcibep and evolocumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 7 Dec 2019to 30 Jan 2023
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
12 sites in 6 countries.
12 sites in 6 countries
India3 sites
CIMS Hospital Pvt. Ltd
Ahmedabad
VMMC & Safdarjung Hospital
Delhi, National Capital Territory of Delhi
G.B. Pant Institute of Postgraduate Medical Education & Research
New Delhi, 110002
United States2 sites
Metabolic & Atherosclerosis Research Center (MARC)
Cincinnati, Ohio, 45227
NorthShore University Health System
Evanston, Illinois, 60201
Israel2 sites
Department of Medicine, Hadassah University Hospital
Jerusalem, 12000
Rabin Medical Center, Beilinson Hospital,
Petah Tikva, 49100
South Africa2 sites
Division of Lipidology, Department of Medicine University of Cape Town
Cape Town, Western Province, 7925
Carbohydrate and Lipid Metabolism Research Unit
Johannesburg, Gauteng, 2193
Turkey (Türkiye)2 sites
Afyonkarahisar Health Sciences University
Afyonkarahisar
Ege University Medical School
Izmir, Bornova, 35040
About this study
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
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
Read the 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).
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- lerodalcibep, evolocumab
- Keywords
- LDL cholesterol, PCSK9 inhibition
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
lerodalcibepDrug
Also called LIB003
PCSK9 inhibitor
evolocumabDrug
Also called Repatha
PCSK9 inhibitor
| Study group | Role | What they receive |
|---|---|---|
| LIB003 (lerodalcibep) | Experimental | 300 mg SC Q4W Receives: Drug: lerodalcibep |
| evolocumab | Active comparator | 420 mg SC Q4W Receives: Drug: evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
Change in serum LDL-C from baseline after 24 weeks
Measured: baseline to 24 weeks on each treatment
Secondary outcomes (1)
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
Measured: baseline to 24 weeks on each treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2019
- Study start7 Dec 2019
- Primary completion12 Sep 2022
- Study completion30 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- open label, randomized, cross-over
- Masking
- None (open label)
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- LIB Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Evan A Stein, MD PhD, Study director, LIB Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04034485
- Sponsor's protocol ID
- LIB003-003
- Status last verified
- Mar 2023
- First submitted
- 22 Jul 2019
- Last updated
- 29 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 39870096
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04034485
- Acronym / sponsor ID
- LIB003-003
- Conditions
- Homozygous Familial Hypercholesterolemia
- Keywords
- LDL cholesterol, PCSK9 inhibition
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- LIB Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- 7 Dec 2019 (actual)
- Primary completion
- 12 Sep 2022 (actual)
- Study completion
- 30 Jan 2023 (actual)
- First posted
- 26 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- open label, randomized, cross-over
| Arm | Type | Receives |
|---|---|---|
| LIB003 (lerodalcibep) | Experimental | Drug: lerodalcibep300 mg SC Q4W |
| evolocumab | Active comparator | Drug: evolocumab420 mg SC Q4W |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| lerodalcibepAlso: LIB003PCSK9 inhibitor | Drug | LIB003 (lerodalcibep) |
| evolocumabAlso: RepathaPCSK9 inhibitor | Drug | evolocumab |
Primary outcome
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24
Time frame: baseline to 24 weeks on each treatment
Change in serum LDL-C from baseline after 24 weeks
1 secondary outcome
The incidence and severity of treatment emergent adverse events (TEAEs)
Time frame: baseline to 24 weeks on each treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2023.
- Derived from this study: PubMed 39870096Raal 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Evan A Stein, MD PhD study director
LIB Therapeutics
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing lerodalcibep and evolocumab in people with Homozygous Familial Hypercholesterolemia, run by LIB Therapeutics LLC at 12 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in India, United States, Israel, South Africa, Turkey (Türkiye) and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent reduction in Low Density Lipoprotein Cholesterol (LDL-C) at week 24 (time frame: baseline to 24 weeks on each treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04034485, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2023.