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

NCT01589237 | Terminated Phase 3 Results

• 2 other identifiers: CLCQ908B23052012-000802-32
• Study Type: interventional
• Target: 38
• Locations: 7 countries
• Sites: 10

Brief Summary

This study was 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).

Conditions

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

Keywords

Familial Chylomicronemia Syndrome (FCS) (HLP type I); LCQ908

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Any Adverse Events, Serious Adverse Events and Death

  52 weeks

Secondary Outcomes (8)

• Changes From Baseline in Triglyceride Levels up to 52 Weeks

  Baseline, Week 12, 24 and 52

• Changes From Baseline in Cholesterol Levels up to 52 Weeks

  Baseline, Week 12, 24 and 52

• Changes From Baseline in HDL and Non HDL Cholesterol Levels up to 52 Weeks

  Baseline, Week 12, 24 and 52

• Changes From Baseline in Glycerol Levels up to 52 Weeks

  Baseline, Week 12, 24 and 52

• Changes From Baseline in Free Fatty Acid Levels up to 52 Weeks

  Baseline, Week 12, 24 and 52

Study Arms (1)

LCQ908

EXPERIMENTAL

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

Interventions

LCQ908 DRUG

LCQ908

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (10)

Novartis Investigative Site

Seatlle, Washington, 98104, United States

Location

Novartis Investigative Site

Chicoutimi, Quebec, G7H 7P2, Canada

Location

Novartis Investigative Site

Ste-Foy, Quebec, G1V4M6, Canada

Location

Novartis Investigative Site

Ouest-Montreal, H2W1R7, Canada

Location

Novartis Investigative Site

Nantes, 44093, France

Location

Novartis Investigative Site

Paris, 75651, France

Location

Novartis Investigative Site

Hamburg, 20246, Germany

Location

Novartis Investigative Site

Meibergdreef 9, 1105 AZ, Netherlands

Location

Novartis Investigative Site

Cape Town, 7925, South Africa

Location

Novartis Investigative Site

Manchester, M13 9NT, United Kingdom

Location

MeSH Terms

Conditions

Familial hyperchylomicronemia syndrome

Interventions

pradigastat

Limitations and Caveats

As the anticipated benefit from the continued participation of patients in 18 month extension (Part B) was not supported by results of the December 2014 interim analysis, Novartis decided to terminate the Part B to be effective as of May 31, 2015.

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

trialandresults.registries@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2012
• First Posted: May 1, 2012
• Study Start: February 1, 2013
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: November 15, 2016
• Results First Posted: November 15, 2016

Record last verified: 2016-09

Locations

DE (1); FR (2); GB (1); NL (1); US (1); CA (3); ZA (1)