Open label, long-term safety and tolerability extension study of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS).
- Conditions
- Familial Chylomicronemia Syndrome (FCS) (HLP Type I)MedDRA version: 17.1Level: LLTClassification code 10020607Term: HyperchylomicronemiaSystem Organ Class: 100000004861Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2012-000802-32-DE
- 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 obtained before any assessment is performed
- Subjects that either discontinue prematurely or complete the CLCQ908B2302 study after 52 weeks or FCS subjects who have previously completed study CLCQ908A2212
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 29
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 13
-Subjects discontinued from the CLCQ908B2302 study for serious, potentially study drug related adverse events
-Subjects from the CLCQ908B2302 study who have developed any other contraindication to participation (for example, renal failure)
- History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
- Preganant 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
- Subjects with type 1 diabetus mellitus or type 2 diabetes mellitus if HbA1c is >=8.5%
- 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 8 weeks prior to randomization. Washout may occur following screening if required
- Glybera [alipogene tiparvovec (AAV1-LPLS447X)] gene therapy exposure within two years prior to screening
- eGFR < 45 ml/min/1.73m2 or history of chronic renal disease
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I);Secondary Objective: To assess changes in lipid and lipoprotein profiles;Primary end point(s): Number of patients with adverse and serious adverse events;Timepoint(s) of evaluation of this end point: up to 52 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - changes in lipid and lipoprotein profiles from baseline up to 52 weeks<br>- changes from baseline in triglyceride levels up to 52 weeks;Timepoint(s) of evaluation of this end point: - baseline, week 12, week 24 and 52<br>- baseline, week 12, week 24 and 52