EUCTR2011-005535-68-DE进行中（未招募）不适用
A randomized, double-blind, placebo controlled study to assess efficacy, safety and tolerability of LCQ908 in subjects with Familial Chylomicronemia Syndrome
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 42
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •- Written informed consent must be performed before any assessment is performed for Period I
- •- Male and female subjects ages at least 18 years of age
- •- Fasting TG = 8.4 mmol/L (750 mg/dL) at Screening
- •- An established diagnosis of Familial Chylomicronemia Syndrome (HLP Type I) confirmed through ultracentrifugation or by documented medical history of a fasting TG = 8.4 mmol/L (750 mg/dL) and by documentation of any of the following at 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 inactivarting antibodies
- •-History of pancreatitis
- •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 42
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •- Although a history of pancreatitis is required, this must be inactive for
- •at least 1 week prior to Screening Visit
- •- Treatment with fish oil preparations within 4 weeks prior to randomization
- •- Treatment with bile acid binding resins (i.e., colesevelam, etc) within 4
- •weeks prior to randomization
- •- Traetment with fibrates within 4 weeks prior to randomization
- •- Glybera [alipogene tiparvovec (AAV1-LPLS447X)] gene therapy
- •exposure within two years prior to screening
- •- History of malignancy of any organ system (other than localised basal
- •cell carcinoma of the skin), treated or untreated, within the past 1 year,
- •regardless of whether there is evidence of local recurrence or
- •metastases.
- •- Any surgical or medical conditions, acute or unstable chronic disease
- •which may, based on investigators opinion, jeopardize the patient in
- •case of participation in the study or might significantly alter the
- •absorption, distribution, metabolism or excretion of the study drug.
- •History of drug or alcohol abuse within the 12 months prior to
- •randomization or evidence of such abuse at screening. Evidence of liver
- •disease or liver injury as indicated by abnormal liver function tests such
- •as AST or ALT, or serum bilirubin.
- •- Use of any other invastigational drugs at the time of screening, or
- •within 30 days or 5 half-lives of enrollment, whichever is longer
- •-eGFR < 30ml/min/1.73m2 or history of chronic renal disease
- •- Participation in any clinical investigation within four (4) weeks prior to
- •initial dosing or longer of required by local regulations or any other
- •limitation of participation based on local regulations
- •- History of hypersensitivity to any of the study drugs or to drugs of
- •similar chemical classes
- •- 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 positive laboratory test.
- •- 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.
研究者
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