A randomized, double-blind, placebo controlled study to assess efficacy, safety and tolerability of LCQ908 in subjects with Familial Chylomicronemia Syndrome
- Conditions
- Familial Chylomicronemia Syndrome (FCS)MedDRA version: 14.1Level: PTClassification code 10051598Term: ChylomicronsSystem Organ Class: 10022891 - InvestigationsTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2011-005535-68-GB
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 42
- 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
- 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
- Treatment 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 investigational 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method