A Placebo-controlled, Double-blind, Randomised, Parallel-group, Long-term Phase III Trial Assessing the Safety and Efficacy of 50 microgram and 100 mircrogram/day of eprotirome in Patients with Heterozygous Familial Hypercholesterolaemia who are on Optimal Standard of Care
- Conditions
- familial hypercholesterolemia100274241001331710003216
- Registration Number
- NL-OMON36068
- Lead Sponsor
- Karo Bio AB
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 160
1) Patients with confirmed HeFH who are equal or above 18 years of age at screening.
2) Patients must also have:
Presence of clinical atherosclerotic disease that confers high risk for CAD events together with an LDL-C above 2 mmol/L at Visit 2; or
Presence of 2 or more risk factors for CVD (other than the HeFH diagnosis) together with an LDL-C above 2.5 mmol/L at Visit 2:
3) Patients should be on an optimal standard of care, defined as being on a stable dose of statin with or without ezetimibe for at least 8 weeks prior to randomisation. If lower doses of rosuvastatin, atorvastatin, or simvastatin or any dose of fluvastatin or pravastatin are used the reasons must be clearly documented.
4) Patients must understand the study procedures and agree to participate in the study by giving written informed consent at Visit 1; and
5) Women must not be pregnant or lactating. Women of childbearing potential must use a medically acceptable form of contraception at least 4 weeks prior to the start of the study and for at least 4 weeks after the patient*s last study visit. Subjects who are on systemic hormonal contraceptives must agree to use an additional method of contraception (barrier method).
Patients should not have other dyslipidaemic or metabolic disorders that could affect the evaluation of eprotirome in HeFH, such as increased serum TG above 4.5 mmol/L, HbA1c above 8.5%, secondary dyslipidaemia or ongoing or planned apharesis.
Patients should also not have cardiac diseases that may interfere with the safety evaluation of eprotirome, such as congestive heart failure (NYHA III and IV), planned percutaneous coronary intervention, coronary artery bypass surgery, uncontrolled hypertension or evidence of cardiac electrophysiologic instability including uncontrolled sick sinus syndrome, sino-atrial or atrioventricular block, ventricular arrhythmias, uncontrolled atrial fibrillation/flutter or uncontrolled supraventricular tachycardia with a ventricular response heart rate or a QTcF above 450 ms prior to randomisation.
No other lipid-lowering medication with the exception of statins and ezetimibe are allowed. Also patients with any medical or surgical condition which might significantly alter the absorption, distribution, metabolism, or excretion of the study medication are excluded.
As eprotirome is a thyroid hormone receptor agonist, patients with disturbed thyroid function, thyrotoxicosis, taking thyroid hormone or anti-thyroid medication are excluded. Drugs known to affect thyroid function tests are also prohibited.
As eprotirome exerts its pharmacological action in the liver, patients with liver dysfunction should be excluded prior to randomisation, such as: AST or ALT or ALP above 1.5 x ULN, total bilirubin >ULN, active hepatobiliary disease, cholestasis, or serologic evidence of past or active hepatitis B or hepatitis C, acquired immune deficiency syndrome, positive serological test for human immunodeficiency virus infection, substantial consumption of alcohol or drug abuse.
Patients with serum creatinine above 160 mmol/L or unexplained serum creatine kinase above 3 x ULN are exluded.
Patients on oral anticoagulant therapy other than vitamin K antagonists and anti-platelet agents are excluded.
Patients with the following diseases are excluded: rheumatoid arthritis; history of cancer, history of or current primary or secondary adrenal insufficiency or any other condition that in the opinion of the investigator confound the evaluation and interpretation of efficacy and or safety data
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method