Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.
This study is currently recruiting participants.
Verified February 2013 by Novartis
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01589237
First received: April 27, 2012
Last updated: February 11, 2013
Last verified: February 2013
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
familial lipoprotein lipase deficiency
Farber lipogranulomatosis
U.S. FDA Resources
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: | January 2013 |
| Estimated Study Completion Date: | March 2014 |
| Estimated Primary Completion Date: | March 2014 (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 to 74 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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.
- Subjects with type 1 diabetes 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 the two years prior to screening.
- eGFR <45 ml/min/1.73m2 or history of chronic renal disease.
Other protocol defined inclusion/exclusion criteria may apply.
Contacts and Locations
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 | Not yet recruiting |
| Portland, Oregon, United States, 97201 | |
| United States, Washington | |
| Novartis Investigative Site | Not yet recruiting |
| Seatlle, Washington, United States, 98104 | |
| Canada, British Columbia | |
| Novartis Investigative Site | Not yet recruiting |
| Vancouver, British Columbia, Canada, V6Z1Y6 | |
| Canada, Quebec | |
| Novartis Investigative Site | Not yet 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 | Not yet recruiting |
| Nantes, France, 44093 | |
| Novartis Investigative Site | Not yet recruiting |
| Paris Cedex 13, France, 75651 | |
| Germany | |
| Novartis Investigative Site | Not yet recruiting |
| Berlin, Germany, 13353 | |
| Novartis Investigative Site | Not yet recruiting |
| Hamburg, Germany, 20246 | |
| Novartis Investigative Site | Not yet recruiting |
| Köln, Germany, 50924 | |
| Netherlands | |
| Novartis Investigative Site | Not yet recruiting |
| Meibergdreef 9, Netherlands, 1105 AZ | |
| South Africa | |
| Novartis Investigative Site | Not yet recruiting |
| Cape Town, South Africa, 7505 | |
| Novartis Investigative Site | Not yet recruiting |
| Cape Town, South Africa, 7925 | |
| Spain | |
| Novartis Investigative Site | Not yet recruiting |
| Malaga, Andalucia, Spain, 29010 | |
| Novartis Investigative Site | Not yet recruiting |
| Sevilla, Andalucía, Spain, 41013 | |
| Novartis Investigative Site | Not yet recruiting |
| Reus, Cataluña, Spain, 43201 | |
| United Kingdom | |
| Novartis Investigative Site | Not yet recruiting |
| Cardiff, United Kingdom, CF14 4XN | |
| Novartis Investigative Site | Not yet 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: | February 11, 2013 |
| 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 May 19, 2013