Eprotirome in Patients with HeFH who are on Optimal Standard of Care

• Research type

  Research Study

• Full title

  A Placebo-controlled, Double-blind, Randomised, Parallel-group, Long-term Phase III Trial Assessing the Safety and Efficacy of 50 µg and 100 µg/day of eprotirome in Patients with Heterozygous Familial Hypercholesterolaemia who are on Optimal Standard of Care

• IRAS ID

  78842

• Contact name

  Dev Datta

• Sponsor organisation

  Karo Bio AB

• Eudract number

  2011-001483-21

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Eprotirome is a thyroid hormone analogue-which reduces cholesterol by it??s action in the liver. It has been shown to lower cholesterol at doses that do not affect thyroid function , the heart or the skeleton. This multi-centre, placebo-controlled, double-blind, randomised, parallel-group, long-term phase III trial will compare the safety and efficacy of eprotirome 50 æg and eprotirome 100 æg versus placebo in terms of reduction in LDL cholesterol from baseline to Week 12 in Heterozygous Familial Hypercholesterolaemia (FH) patients with coronary artery disease (CAD), or who are at high risk for CAD, and who are on optimal therapy consisting of a statin with or without ezetimibe. FH is an inherited disorder,affecting about 1 in 500 of the population, where those affected have very high levels of cholesterol from birth.The study will be performed in approximately 70 study centres throughout the world. Approximately 630 men and women, aged 18 years old or older will participate in the study. Patients will be made aware of the study by individual investigators of the participating institutions. Each patient will be evaluated for approximately 2 years and 18 weeks. Some patients in the study will undergo extra safety assessments such as monitoring of heart function and bone density. In Phase II studies, pronounced and clinically relevant reduction in cardiovascular risk factors in the blood (such as cholesterol levels) were documented in subjects given eprotirome as an add-on to statin or ezetimibe treatment. Patients will be carefully monitored throughout the study.The proposed research has been designed and will be funded by Karo Bio AB.

• REC name

  Wales REC 3

• REC reference

  11/WA/0142

• Date of REC Opinion

  28 Jul 2011

• REC opinion

  Further Information Favourable Opinion