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

NCT ID: NCT01589237

Last Updated: 2016-11-15

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 38 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-02-28
• Study Completion Date: 2015-07-31

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).

Detailed Description

This study was an 52 weeks open label extension starting at the lowest treatment dose used in CLCQ908B2302/NCT01514461 (i.e., 10 mg) with optional up-titrations, to evaluate the overall long-term safety and tolerability of LCQ908 in patients with Familial Chylomicronemia Syndrome, who either discontinued from the CLCQ908B2302/NCT01514461 study (due to tolerability issues) or completed the CLCQ908B2302/NCT01514461 study after 52 weeks. In addition, patients who had previously completed study CLCQ908A2212/NCT01146522 were eligible to participate.

Following Protocol amendment 2, the original 52 week duration of this study (CLCQ908B2305) became Part A of LCQ908B2305 and a 78 week extension became Part B. However, following Protocol amendment 3, Part B was ended at the same time as the last patient of Part A completed 52 weeks. The reason for termination of Part B was the findings from the December 2014 interim analysis which suggested that the size of benefit that was anticipated from continued participation of patients in the 18 month extension trial (Part B) no longer supported trial extension beyond Part A.

Conditions

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

Keywords

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

LCQ908

Intervention Type: DRUG

Interventions

LCQ908

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Seatlle, Washington, United States

Novartis Investigative Site

Chicoutimi, Quebec, Canada

Novartis Investigative Site

Ste-Foy, Quebec, Canada

Novartis Investigative Site

Ouest-Montreal, , Canada

Novartis Investigative Site

Nantes, , France

Novartis Investigative Site

Paris, , France

Novartis Investigative Site

Hamburg, , Germany

Novartis Investigative Site

Meibergdreef 9, , Netherlands

Novartis Investigative Site

Cape Town, , South Africa

Novartis Investigative Site

Manchester, , United Kingdom

Countries

Spain; United States; Canada; France; Germany; Netherlands; South Africa; United Kingdom

Other Identifiers

2012-000802-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CLCQ908B2305

Identifier Type: -

Identifier Source: org_study_id