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

ovartis0 个研究点目标入组 4 人开始时间: 待定最近更新: 2024年5月6日

适应症

试验速览

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•1) Male and female subjects 18 years of age or above3) Fasting TG * 8.4 mmol/L at Screening
•2) Fasting TG * 8.4 mmol/L at Screening
•3) An established diagnosis of FCS (HLP Type I) confirmed through ultracentrifugation or by documented medical history of a fasting TG * 8.4 mmol/L and by documentation of any of the following at Screening or during the Screening Period:
•a) Confirmed homozygote or compound heterozygote for known loss-of-function mutations in Type I-causing genes (such as LPL, apoCII, GPIHBP1, or LMF1)
•b) Post heparin plasma LPL activity of * 20% of normal
•c) Confirmed presence of LPL inactivating antibodies
•4) History of pancreatitis.

•1) Although a history of pancreatitis is required, this must be inactive for at least 1 week prior to the Screening Visit.
•2) Treatment with fish oil preparations within 4 weeks prior to randomization.
•3) Treatment with bile acid binding resins (i.e., colesevelam, etc) within 4 weeks prior to randomization.
•4) Treatment with fibrates within 4 weeks prior to randomization.
•5) eGFR <30 ml/min/1.73m2 or history of chronic renal disease
•6) Participation in any clinical investigation within 4 weeks prior to initial dosing or longer if required by local regulations, or any other limitation of participation based on local regulations.
•7) Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive HCG laboratory test.
•8) Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 100 days after discontinuation of investigational study drug.

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