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Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: April 27, 2012
Last updated: March 16, 2016
Last verified: March 2016
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).

Condition Intervention Phase
Familial Chylomicronemia Syndrome (FCS) (HLP Type I)
Drug: LCQ908
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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.

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Number of patients wtih Adverse and Serious Adverse Events [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
    AE/SAE monitoring will occur. Gastrointestinal AEs and any symptoms of phototoxicity; events suggestive or diagnostic of acute pancreatitis and will be adjudicated.

Secondary Outcome Measures:
  • Changes in lipid and lipoprotein profiles from baseline up to 52 weeks [ Time Frame: Baseline, Week 12, 24 and 52 ] [ Designated as safety issue: No ]
    Fasting blood samples will be collected by direct venipuncture or an indwelling cannula to evaluate the drug effect on lipid/lipoprotein profiles.

  • Changes from baseline in triglyceride levels up to 52 weeks [ Time Frame: Baseline, Week 12, 24 and 52 ] [ Designated as safety issue: No ]
    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.

Enrollment: 38
Study Start Date: February 2013
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LCQ908
Patients initiated at 10 mg/day. After at least 8 weeks of treatment with a dose, optional up-titration to the next possible dose will be allowed. One down titration allowed from the highest dose attained.
Drug: LCQ908


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01589237

United States, Washington
Novartis Investigative Site
Seatlle, Washington, United States, 98104
Canada, Quebec
Novartis Investigative Site
Chicoutimi, Quebec, Canada, G7H 7P2
Novartis Investigative Site
Ste-Foy, Quebec, Canada, G1V4M6
Novartis Investigative Site
Ouest-Montreal, Canada, H2W1R7
Novartis Investigative Site
Nantes, France, 44093
Novartis Investigative Site
Paris Cedex 13, France, 75651
Novartis Investigative Site
Hamburg, Germany, 20246
Novartis Investigative Site
Meibergdreef 9, Netherlands, 1105 AZ
South Africa
Novartis Investigative Site
Cape Town, South Africa, 7925
United Kingdom
Novartis Investigative Site
Manchester, United Kingdom, M13 9NT
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT01589237     History of Changes
Other Study ID Numbers: CLCQ908B2305  2012-000802-32 
Study First Received: April 27, 2012
Last Updated: March 16, 2016
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Netherlands: European Medicines Agency
France : European Medicines Agency
Germany: European Medicines Agency
Spain:European Medicines Agency
United Kingdom:European Medicines Agency
South Africa: Medicines Control Council

Keywords provided by Novartis:
Familial Chylomicronemia Syndrome (FCS) (HLP type I)

Additional relevant MeSH terms:
Hyperlipoproteinemia Type I
Pathologic Processes
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases processed this record on October 25, 2016