Efficacy Study of Diazoxide Choline to Treat Hypertriglyceridemia

A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Diazoxide Choline Controlled-Release Tablet (DCCR) in Subjects Without Diabetes Mellitus Having Very High Fasting Triglyceride Levels, With Double-Blind DCCR-Fenofibrate Combination Extension

Once a day oral administration with DCCR helps lower triglycerides

Study Overview

• Status: Completed
• Conditions: Dyslipidemia; Hypertriglyceridemia
• Intervention / Treatment: Drug: Diazoxide Choline Controlled-Release Tablet; Drug: Placebo

Detailed Description

The population will consist of Statin-naive and Statin-treated subjects, all without diabetes mellitus, with fasting triglyceride levels in the range of ≥ 500 mg/dL and < 1500 mg/dL.

Subjects will be randomly assigned to 1 of 2 treatment groups: DCCR and Placebo

Approximately 44 subjects will be enrolled in the study and stratified by Statin use at a 1:1 ratio in each treatment group,

• Study Type: Interventional
• Enrollment (Actual): 44
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

INCLUSION CRITERIA:

Fasting triglycerides

• Difference between Visit 3 (7 days prior to Baseline Visit) and Visit 4 (3 days prior to Baseline Visit) ≤ 60% (compared to the higher value of Visit 3 or Visit 4)
• Run-in Triglycerides* ≥ 500 mg/dL and < 1500 mg/dL *Run-in Triglyceride is defined as the average fasting triglycerides for Visit 3 (7 days prior to Baseline Visit) and Visit 4 (3 days prior to Baseline Visit).

Statin use • Either Statin-naive

• Must not be on statin at Screening and remaining as such during the Run-in/Washout Period and throughout the study

  • Or Statin-treated

• Must be on Lipitor 20 mg initiated at the start of the Run-in/Washout Period and continue throughout the study

Medication washout

• All subjects must be willing to undergo washout of all other lipid-lowering medications

Glycemic status

• Fasting glucose < 126 mg/dL at Screening Visit
• HbA1c < 6.5% at Screening Visit

EXCLUSION CRITERIA:

Medications: recent, current, anticipated

• Administration of investigational drugs within 1 month prior to Screening Visit
• Thyroid hormones or preparations within 1 month prior to Screening Visit (except in subjects on stable dose of replacement therapy for at least 1 month)
• Thiazide diuretics within 2 weeks prior to Screening Visit
• Discontinuation of beta-blockers within 1 month prior to Screening Visit or planned discontinuation of beta-blocker therapy
• Anticipated requirement for use of prohibited concomitant medications

History of allergic reaction or significant intolerance to:

• Diazoxide
• Thiazides
• Sulfonamides
• Fenofibrate or fenofibric acid derivatives

Lifestyle changes

• Subjects intending to change exercise habits, quit smoking and/or quit alcohol use during the entire study

Specific diagnoses, medical conditions and history

• Known type I or III hyperlipidemia
• Known type 1 DM
• Current diagnosis of type 2 DM
• Any other clinically significant endocrine, cardiovascular, pulmonary, neurological, psychiatric, hepatic, gastrointestinal, hematological, renal, or dermatological disease interfering with the assessments of the study medications, according to the Investigator

Specific laboratory test results

• Any relevant biochemical abnormality interfering with the assessments of the study medications

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: DCCR; DCCR Treatment with 290 mg Diazoxide Choline	Drug: Diazoxide Choline Controlled-Release Tablet; DCCR 290 mg once a day for 126 days
Placebo Comparator: Placebo; Placebo matching DCCR	Drug: Placebo; Placebo matching DCCR

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Triglyceride-lowering efficacy of DCCR	Fasting triglycerides (percent change from Baseline to Day 84)	84 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Improvement in other lipid profiles with DCCR	Apolipoprotein B (Apo B) (percent change from Baseline to Day 84) Fasting non-HDL cholesterol (percent change from Baseline to Day 84)	84 days

Collaborators and Investigators

• Sponsor: Essentialis, Inc.
• Collaborators: Medpace, Inc.
• Investigators: Study Director: Alain Baron, M.D., Essentialis, Inc.

Study record dates

Study Major Dates

• Study Start: October 1, 2010
• Primary Completion (Actual): September 1, 2011
• Study Completion (Actual): December 1, 2011

Study Registration Dates

• First Submitted: September 29, 2010
• First Submitted That Met QC Criteria: September 29, 2010
• First Posted (Estimate): September 30, 2010

Study Record Updates

• Last Update Posted (Estimate): January 10, 2014
• Last Update Submitted That Met QC Criteria: January 9, 2014
• Last Verified: January 1, 2014

More Information

Terms related to this study

• Keywords: fenofibrate; triglycerides; statin; Apo B; non-HDL
• Additional Relevant MeSH Terms: Metabolic Diseases; Lipid Metabolism Disorders; Hyperlipidemias; Dyslipidemias; Hypertriglyceridemia; Molecular Mechanisms of Pharmacological Action; Antihypertensive Agents; Vasodilator Agents; Antimetabolites; Gastrointestinal Agents; Hypolipidemic Agents; Lipid Regulating Agents; Nootropic Agents; Lipotropic Agents; Diazoxide; Choline
• Other Study ID Numbers: CT013