Familial Chylomicronemia Syndrome (FCS) (HLP Type I) Clinical Trial
Official title:
An Open Label, 52-week, Safety and Tolerability Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.
This study is 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).
| Status | Completed |
| Enrollment | 38 |
| Est. completion date | July 2015 |
| Est. primary completion date | July 2015 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: 1. Written informed consent must be obtained before any assessment is performed. 2. Subjects that either discontinue prematurely or complete the CLCQ908B2302 study after 52 weeks or FCS subjects who have previously completed study CLCQ908A2212. Exclusion Criteria: 1. Subjects discontinued from the CLCQ908B2302 study for serious, potentially study drug related adverse events. 2. Subjects from the CLCQ908B2302 study who have developed any other contraindication to participation (for example, renal failure) 3. 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. 4. 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. 5. Subjects with type 1 diabetes mellitus or type 2 diabetes mellitus if HbA1C is = 8.5%. 6. Treatment with fish oil preparations within 4 weeks prior to randomization. 7. Treatment with bile acid binding resins (i.e., colesevelam, etc) within 4 weeks prior to randomization. 8. Treatment with fibrates within 8 weeks prior to randomization. Washout may occur following screening if required. 9. Glybera [alipogene tiparvovec (AAV1-LPLS447X )] gene therapy exposure within the two years prior to screening. 10. eGFR <45 ml/min/1.73m2 or history of chronic renal disease. Other protocol defined inclusion/exclusion criteria may apply. |
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Canada | Novartis Investigative Site | Chicoutimi | Quebec |
| Canada | Novartis Investigative Site | Ouest-Montreal | |
| Canada | Novartis Investigative Site | Ste-Foy | Quebec |
| France | Novartis Investigative Site | Nantes | |
| France | Novartis Investigative Site | Paris Cedex 13 | |
| Germany | Novartis Investigative Site | Hamburg | |
| Netherlands | Novartis Investigative Site | Meibergdreef 9 | |
| South Africa | Novartis Investigative Site | Cape Town | |
| United Kingdom | Novartis Investigative Site | Manchester | |
| United States | Novartis Investigative Site | Seatlle | Washington |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
United States, Canada, France, Germany, Netherlands, South Africa, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of patients wtih Adverse and Serious Adverse Events | AE/SAE monitoring will occur. Gastrointestinal AEs and any symptoms of phototoxicity; events suggestive or diagnostic of acute pancreatitis and will be adjudicated. | 52 weeks | Yes |
| Secondary | Changes in lipid and lipoprotein profiles from baseline up to 52 weeks | Fasting blood samples will be collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipid/lipoprotein profiles. | Baseline, Week 12, 24 and 52 | No |
| Secondary | Changes from baseline in triglyceride levels up to 52 weeks | Blood samples will be collected for a fasting lipid panel, including total triglycerides. Lipid measurements should be collected after a 12 hour (overnight) fast. The maintenance of effect will be assessed on triglyceride levels during continued therapy with LCQ908 for up to 52 weeks in the full analysis set. | Baseline, Week 12, 24 and 52 | No |