Effect of LCQ908 in Subjects with Familial Chylomicronemia Syndrome

  • Research type

    Research Study

  • Full title

    A randomized, double-blind, placebo controlled study to assess the efficacy, safety and tolerability of LCQ908 in subjects with Familial Chylomicronemia Syndrome.



  • Contact name

    Handrean Soran

  • Sponsor organisation

    Novartis Pharma Services AG

  • Eudract number


  • Research summary

    The purpose of this study is to determine whether a new investigational drug, LCQ908 is effective and safe in lowering triglycerides (a type of fat) in subjects with a rare genetic disorder, Familial Chylomicronaemia Syndrome (FCS). FCS leads to very high lipid levels causing an increased risk of acute pancreatitis (sudden swelling/inflammation of teh pancreas), which can be severe and life threatening. This study aims to compare two dosing levels of LCQ908 with placebo (dummy drug of inactive substance). The subjects will enter the study on a strict very low fat diet, which is currently the only available treatment for this condition. They will take the study drug (or placebo) in tablet form once daily for 52 weeks. This will involve taking 2 or 3 tablets daily, depending on dose. It will be possible after the first 12 weeks of treatment to reduce the active dose of drug if it is not well tolerated. The study is a randomized double blind design, so neither the subject nor the investigator will know which dose the subject is on or whether the subject has been allocated placebo. In total a patient??s involvement will last 13-14 months and will involve 14 study visits for study related procedures.

  • REC name

    North West - Greater Manchester Central Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    19 Jul 2012

  • REC opinion

    Further Information Favourable Opinion