Trial record 1 of 8 for:    "familial lipoprotein lipase deficiency"
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Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01589237
First received: April 27, 2012
Last updated: June 25, 2014
Last verified: June 2014
  Purpose

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: Allocation: Non-Randomized
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.


Estimated Enrollment: 42
Study Start Date: February 2013
Estimated Study Completion Date: November 2016
Estimated Primary Completion Date: November 2016 (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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT01589237

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

Locations
United States, Oregon
Novartis Investigative Site Withdrawn
Portland, Oregon, United States, 97201
United States, Washington
Novartis Investigative Site Recruiting
Seatlle, Washington, United States, 98104
Canada, British Columbia
Novartis Investigative Site Withdrawn
Vancouver, British Columbia, Canada, V6Z1Y6
Canada, Quebec
Novartis Investigative Site Recruiting
Chicoutimi, Quebec, Canada, G7H 7P2
Novartis Investigative Site Recruiting
Ste-Foy, Quebec, Canada, G1V4M6
Canada
Novartis Investigative Site Not yet recruiting
Ouest-Montreal, Canada, H2W1R7
France
Novartis Investigative Site Recruiting
Nantes, France, 44093
Novartis Investigative Site Not yet recruiting
Nantes, France, 44093
Novartis Investigative Site Not yet recruiting
Paris Cedex 13, France, 75651
Germany
Novartis Investigative Site Withdrawn
Berlin, Germany, 13353
Novartis Investigative Site Not yet recruiting
Hamburg, Germany, 20246
Novartis Investigative Site Withdrawn
Köln, Germany, 50924
Netherlands
Novartis Investigative Site Recruiting
Meibergdreef 9, Netherlands, 1105 AZ
South Africa
Novartis Investigative Site Withdrawn
Cape Town, South Africa, 7505
Novartis Investigative Site Recruiting
Cape Town, South Africa, 7925
Spain
Novartis Investigative Site Withdrawn
Malaga, Andalucia, Spain, 29010
Novartis Investigative Site Withdrawn
Sevilla, Andalucia, Spain, 41013
Novartis Investigative Site Withdrawn
Reus, Cataluña, Spain, 43201
United Kingdom
Novartis Investigative Site Withdrawn
Cardiff, United Kingdom, CF14 4XN
Novartis Investigative Site Recruiting
Manchester, United Kingdom, M13 9NT
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01589237     History of Changes
Other Study ID Numbers: CLCQ908B2305, 2012-000802-32
Study First Received: April 27, 2012
Last Updated: June 25, 2014
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)
LCQ908

Additional relevant MeSH terms:
Hyperlipoproteinemia Type I
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on July 20, 2014