Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease MedDRA version: 20.0 Level: LLT Classification code 10057079 Term: Heterozygous familial hypercholesterolemia System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 10010331 - Congenital, familial and genetic di
Conditions
Sponsors
LIB Therapeutics, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient has received study drug through the end of study (EOS) with a complete EOS Visit in 1 of the Phase 3 base studies, LIB003-003, -004, -005, -006, -008, -011, and -012, without SAEs related to LIB003; 2. Patient has the provision of written and signed informed consent prior to any study-specific procedure; 3. Female patients of childbearing potential must continue using a highly effective form of birth control if sexually active and have a negative urine pregnancy test on Day 1 prior to dosing;; o Note: Highly effective methods of birth control include refraining from heterosexual sexual intercourse during the entire period of risk, birthcontrol pills or patches, intrauterine devices (IUDs), sexual activity with a male partner who has had a vasectomy, or IUD, oral, implantable, or injectable contraceptives. Menopause is defined as 1 year of spontaneous and continuous amenorrhea in a female =55 years old, or 1 year of spontaneous and continuous amenorrhea with a folliclestimulating hormone (FSH) level >40 IU/L (or according to the definition of "postmenopausal range" for the laboratory involved) in a female =65 years) yes F.1.3.1 Number of subjects for t
Exclusion criteria
Exclusion criteria: 1. Failure to receive study drug through the EOS or to complete the EOS Visit in the Phase 3 base study (LIB003-003, -004, -005, -006, -008, -011, or -012) and/or had an SAE that was considered related to study drug during the Phase 3 base study; 2. Development since the final visit in the Phase 3 base study (LIB003-003, -004, -005, -006, 008, -011, or-012) of any concomitant clinical condition or acute and/or unstable systemic disease compromising patient inclusion, at the discretion of the Investigator, including but not limited to, the following: a history or presence of clinically significant pulmonary, hepatic, gallbladder or biliary tract, hematologic, gastrointestinal, endocrine (excluding diabetes), immunologic, dermatologic, neurologic, or psychiatric disease, which in the Investigator's opinion would not be suitable for the study from a patient safety consideration or could interfere with the results of the study; 3. Use of prohibited oral lipid lowering agents, PCSK9 mAbs, mipomersen, lomitapide, gemfibrozil, or bempedoic acid, started since completion of the base study; o Note: Use of lomitapide is not approved for, and will be prohibited in, patients from LIB003-004, -005, -006, -008, -011, and -012. o Note: Use of bempedoic acid is not approved for, or will be prohibited in, patients from LIB003-003, -008, -011, and -012. 4. Not available for protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge; 5. Has any other findings since the completion of the base study which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study; or 6. Is an employee or family member of the Investigator or study site personnel.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objectives of this study are to assess the long-term safety, tolerability, and efficacy after 48 and 72 weeks with monthly (Q4W [= 31 days]) dosing of LIB003 300 mg administered subcutaneously (SC). The study includes patients at very-high risk for CVD or at high risk for CVD (including HoFH and HeFH) on a stable diet and maximally tolerated oral LDL-C lowering drug therapy who completed a LIB003 Phase 3 base study.;Secondary Objective: There are not secondary objectives in this study. ;Primary end point(s): Efficacy Endpoints • LDL-C change (absolute and percent) compared to original baseline LDL-C at Day 1 in the base study calculated by both Friedewald and Hopkins formulas and preparative ultracentrifugation at Weeks 48 and 72 for patients entering the OLE directly from studies LIB003-004, -005, -006, -008, -011, and -012. For patients entering from the LIB003-011 and -012 studies, LDL-C efficacy will also be assessed at Week 12 and compared to the Week 12 or Day 270 of the base studies, respectively; • LDL-C change (absolute and percent) compared to original baseline LDL-C at Day 1 in the base study calculated by both Friedewald and Hopkins formulas and preparative ultracentrifugation at Weeks 48 and 72 for patients entering the OLE directly from study LIB003-003; • Effects of LIB003 at Weeks 48 and 72 on serum lipids, including TC, HDL-C, non–HDL-C, VLDL-C, and TG; • Effects of LIB003 at Weeks 48 and 72 on apo B and Lp(a) serum concentrations. For patients entering from the LIB003-011 and -012 studies, effects on apo B and Lp(a) will also be assessed at Week 12 compared to Week 12 or Day 270 of the base studies, respectively; • Effects of LIB003 at Weeks 48 and 72 on serum unbound (free) PCSK9 concentration; and • The percentage of patients achieving current ESC/EAS guidelines. Immunogenicity Endpoints Anti-LIB003 antibodies will be initially measured at Week 72/Early Termination [ET]. Other visits including Weeks 12, 24, 36, 48, an | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): There are not secondary endpoints in this study. ;Timepoint(s) of evaluation of this end point: Not applicable. There are not secondary endpoints in this study. | — |
Countries
Canada, France, Germany, India, Israel, New Zealand, Norway, South Africa, Spain, Türkiye, United Kingdom, United States
Contacts
Public ContactLIB clinical trials
LIB Therapeutics, LLC
Outcome results
None listed