Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease.
- Conditions
- Patients with elevated LDL-C, with homozygous familial hypercholesterolemia (HoFH) and heterozygous familialhypercholesterolemia (HeFH), cardiovascular disease, or at high risk for cardiovascular disease.MedDRA version: 20.0Level: LLTClassification code 10057079Term: Heterozygous familial hypercholesterolemiaSystem Organ Class: 100000004850MedDRA version: 20.0Level: LLTClassification code 10057080Term: Homozygous familial hypercholesterolemiaSystem Organ Class: 100000004850MedDRA version: 20.1Level: LLTClassification code 10007648Term: Cardiovascular disease, unspecifiedSystem Organ Class: 100000004849Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2020-004394-49-ES
- Lead Sponsor
- IB Therapeutics, LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 2000
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. Women 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, birth control 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 follicle-stimulating hormone (FSH) level >40 IU/L (or according to the definition of postmenopausal range” for the laboratory involved) in a female <55 years old unless the patient has undergone bilateral oophorectomy. Birth control should be maintained for 60 days after the last dose of study drug (ie, 30 days after the last study visit).
oNote: WOCBP will also have a pregnancy test at every visit to the clinic for the duration of the study.
4. Male patients will either be surgically sterile or agree to continue to use the following forms of contraception if their partner is of childbearing potential and not using a highly effective form of birth control as defined in Inclusion Criterion #3: male or female condom with spermicide and a female partner who is sterile or who agrees to use the following contraceptives; diaphragm or cervical cap with spermicide; or IUD, oral, implantable, or injectable contraceptives;
5. Male patients must refrain from sperm donation until 90 days following the last dose of study drug;
6. Patient is willing to maintain appropriate diet and stable dose of current LLT, including statins, ezetimibe, bile acid sequestrants, niacin, lomitapide, bempedoic acid, bezafibrate or fenofibrate, and/or OM-3 compounds; and
o Note: Use of lomitapide will be allowed in patients from LIB003-003.
o Note: Use of bempedoic acid will be allowed in patients from LIB003-004, -005, and -006.
o Note: Patients still requiring apheresis may add the procedure after Week 12.
7. Patient is considered by the Investigator to be otherwise healthy, based on medical history review, a defined complete physical examination, as well as vital sign measurements, ECGs, and laboratory test results in the base trial.
Are the trial subjects under 18? yes
Number of subjects for this age range: 17
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1250
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 733
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, or -011) 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 new 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method