A clinical study on patients suffering from high level cholesterols. The aim is to evaluate safety and efficacy of a new medicinal drug.

• Conditions: Heterozygous Familial Hypercholesterolaemia; MedDRA version: 14.0Level: LLTClassification code 10057099Term: Heterozygous familial hypercholesterolaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2011-001483-21-AT
• Lead Sponsor: Karo Bio AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-05-24
• Last Update Posted: 16 October 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 1050

Inclusion Criteria

1)Patients with confirmed HeFH who are 18 years of age or older, understand the study procedures and agree to participate in the study by giving written informed consent at Visit 1 and must have:
a) Presence of clinical atherosclerotic disease that confers high risk for CAD events together with an LDL-C >2 mmol/L (>80 mg/dL) at Visit 2, based on one or more of the following; or
b) Presence of at least 2 risk factors for CVD (other than the HeFH diagnosis) together with an LDL-C >2.5 mmol/L (>100 mg/dL) at Visit 2:
2) Patients should be on an optimal standard of care, defined as being on a stable dose of statin (at least half of the maximum dose of either rosuvastatin, atorvastatin, or simvastatin) 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.
3) 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).
4) Diabetes mellitus (Type 1 and Type 2), for Type 2 (only metformin, sulfonylureas, alpha-glucosidase inhibitors or DPP-4s as antidiabetic medications are allowed)
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1050
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1) Other dyslipidaemic or metabolic disorders that could affect the evaluation of eprotirome in HeFH, such as increased serum TG >4.5 mmol/L, HbA1c >8.5%, secondary dyslipidaemia or ongoing or planned apharesis
2) 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, unstable angina pectoris, myocardial infarction, uncontrolled hypertension (defined as systolic blood pressure equal to or more than 160 mmHg or diastolic blood pressure equal to or more than 100 mmHg) 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 (>100 bpm) or a QTcF >450 ms
3) Previous stroke (includes definite or presumed cerebral ischaemia/infarction)
4) No other lipid-lowering medication with the exception of statins and ezetimibe are allowed
5) Patients with disturbed thyroid function, thyrotoxicosis, taking thyroid hormone or anti-thyroid medication are excluded
6) Drugs known to affect thyroid function tests are also prohibited
7) Patients with liver dysfunction such as: AST or ALT or ALP >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 HIV, substantial consumption of alcohol or drug abuse
8) Patients with serum creatinine >160 mmol/L or unexplained serum creatine kinase >3 x ULN
9) Patients on oral anticoagulant therapy other than vitamin K antagonists and anti-platelets agents
10) 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 clinical trial of medicinal product
• Study Design: Not specified