A 12-Week Study to Assess the Efficacy Safety and Tolerability of Gemcabene in Subjects With Severe Hypertriglyceridemia

NCT02944383 | Completed Phase 2 Results

• 1 other identifier: GEM-401
• Study Type: interventional
• Target: 91
• Locations: 2 countries
• Sites: 49

Brief Summary

A 12-Week, Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy Safety and Tolerability of Gemcabene in Subjects with Severe Hypertriglyceridemia (INDIGO-1)

Conditions

Severe Hypertriglyceridemia; Mixed Dyslipidaemia

Keywords

Hypertriglycerides

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline to End of Study (EOS) in Fasting Serum Triglycerides (TG)

  EOS was defined as the average of the week 10 and week 12 values. If either the week 10 or week 12 value was missing, then the single value (week 10 or week 12) was used. Completely missing values (both week 10 and week 12) were imputed using last observation carried forward (LOCF).

  Baseline, EOS (average of week 10 and 12)

Secondary Outcomes (49)

• Percent Change From Baseline in Fasting Serum TG

  Baseline, Weeks 2, 6, 10 and 12

• Change From Baseline in Fasting Serum TG

  Baseline, Weeks 2, 6, 10, 12 and EOS (average of week 10 and 12)

• Percent Change From Baseline in TC

  Baseline, Weeks 2, 6, 10, 12 and EOS (average of week 10 and 12)

• Change From Baseline in TC

  Baseline, Weeks 2, 6, 10, 12 and EOS (average of week 10 and 12)

• Percent Change From Baseline in Non-HDL-C

  Baseline, Weeks 2, 6, 10, 12 and EOS (average of week 10 and 12)

Study Arms (3)

Gemcabene 300 mg

EXPERIMENTAL

Participants received 300 mg Gemcabene orally, once daily for 12 weeks.

Drug: Gemcabene

Gemcabene 600 mg

EXPERIMENTAL

Participants received 600 mg Gemcabene orally, once daily for 12 weeks.

Drug: Gemcabene

Placebo

PLACEBO COMPARATOR

Participants received matching placebo orally, once daily for 12 weeks.

Drug: Placebo

Interventions

Gemcabene DRUG

Gemcabene tablets administered orally once daily, for 12 weeks.

Gemcabene 300 mg; Gemcabene 600 mg

Placebo DRUG

Placebo tablets administered orally once daily, for 12 weeks.

Placebo

Eligibility Criteria

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

You may qualify if:

• Subjects who meet all of the following criteria will be eligible to participate in the study:
• Provision of written and signed informed consent (by subject or legal guardian) prior to any study-specific procedure;
• Male or female (neither pregnant or lactating) ≥18 years of age at time of consent;
• Women of child-bearing potential must have a negative serum pregnancy test at the Screening Visit and negative urine dipstick on Study Day 1 prior to dosing in order to qualify for the study. Women who are surgically sterile or are clinically confirmed to be post-menopausal (i.e., documented amenorrhea for ≥ 1 year in the absence of other biological or physiological causes) are not considered to be of child-bearing potential;
• Women of child-bearing potential must agree to use acceptable methods of contraception throughout the duration of the study and for 30 days after the last dose of study drug. For this study, double-barrier contraception is required.
• Currently on a self-reported, stable, low-fat, low-cholesterol diet in combination with stable statins with or without ezetimibe (10 mg QD) for at least 12 weeks prior to the Screening Visit;
• Mean fasting TG value ≥ 500 mg/dL to \< 1500 mg/dL (with the higher value no more than 50% greater than the lower value) from the S1 and S2 Visits (or alternatively S2 and S3);
• Physical examination, including vital signs, that is within normal limits or clinically acceptable to the Investigator;
• Weight ≥ 50 kg; with a body mass index (BMI) ≤ 45 kg/m²; and
• Subjects with Type 2 diabetes who take anti-diabetes pharmacologic therapy must be on a stable a regimen for at least 3 months, with no planned changes in medications for the study duration.

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from participation in the study:
• Known and previously documented homozygous genetic deficiencies (LPL, ApoC-II, ApoC-III, ApoA-V, GPIHBP1, or LMF1);
• History of pancreatitis within the last 6 months prior to screening (Visit S1);
• History of bariatric surgery; symptomatic gallstone disease, unless treated with cholecystectomy;
• Abnormal liver function test at the Pre-Screening Visit or any of the Screening Visits (aspartate aminotransferase or alanine aminotransferase \> 2 × the upper limit of normal \[ULN\], total bilirubin \> 1.5 × ULN, or alkaline phosphatase \> 2 × ULN based on appropriate age and gender normal values). Subjects with bilirubin \> 1.5 × ULN and history of Gilbert's syndrome may be included; reflexive direct bilirubin testing will be used to confirm Gilbert's syndrome;
• Active liver disease (e.g., cirrhosis, alcoholic liver disease, hepatitis B \[HBV\], hepatitis C \[HCV\], autoimmune hepatitis, liver failure, liver cancer), history of liver transplant, known diagnosis of human immunodeficiency virus (HIV), or acquired immune deficiency virus;
• Moderate to severe renal insufficiency defined as an estimated GFR \< 60 mL/min/1.73 m2 (calculated using The Chronic Kidney Disease Epidemiology Collaboration equation) at the Pre-Screening Visit or at any of the Screening Visits;
• Abnormal urinalysis (proteinuria greater than trace or any male or non-menstruating female with greater than trace hematuria), confirmed by reflexive urine protein:creatinine ratio testing;
• Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) below the lower limit of normal or \> 1.5 × ULN, respectively, based on results from the Pre-Screening Visit or the Screening Visit. If controlled, treatment should be stable for at least 3 months prior to the Screening Visit;
• Type 1 diabetes mellitus or uncontrolled type 2 diabetes mellitus HbA1c value ≥8.5% based on results from the Pre-Screening Visit or the Screening Visit), or any diabetic subject taking a thiazolidinedione (e.g., pioglitazone, rosiglitazone);
• New York Heart Association Class III or IV heart failure (see Appendix C);
• Myocardial infarction, severe or unstable angina pectoris, coronary angioplasty, coronary artery bypass graft, or other major cardiovascular events resulting in hospitalization within 3 months of the Screening Visit (S1). Subjects with adequately treated stable angina, per Investigator assessment, may be included;
• Uncontrolled cardiac arrhythmia or prolonged QT on the Screening Visit or Study Day 1 prior to dosing ECG (QTcF \> 450 msec for men and \>470 msec for women) or known family history of prolonged QT or unexplained sudden cardiac death;
• Uncontrolled hypertension, defined as sitting systolic blood pressure ≥ 180 mmHg or diastolic blood pressure ≥ 110 mmHg, and confirmed by repeat measurement;
• Currently receiving cancer treatment(s) or, in the Investigator's opinion, at risk of relapse for recent cancer;

Sponsors & Collaborators

• NeuroBo Pharmaceuticals Inc.: lead

Study Sites (49)

Appalachian Research Associates

Fort Payne, Alabama, 35967, United States

Location

Del Sol Research Mangagement, LLC

Tucson, Arizona, 85710, United States

Location

Westside Medical Associates of Los Angeles

Beverly Hills, California, 90211, United States

Location

Hope Clinical Research

Canoga Park, California, 91303, United States

Location

SC Clinical Research

Garden Grove, California, 92844, United States

Location

National Research Institute

Huntington Park, California, 90255, United States

Location

National Research Institute

Los Angeles, California, 90057, United States

Location

Paradigm Clinical Research

San Diego, California, 92117, United States

Location

Paradigm Research

Wheat Ridge, Colorado, 80033, United States

Location

Excel Medical Research

Boca Raton, Florida, 33434, United States

Location

Meridien Research

Bradenton, Florida, 34201, United States

Location

Direct Helpers Research Center

Hialeah, Florida, 33012, United States

Location

Indago Research and Health Center

Hialeah, Florida, 33012, United States

Location

Jacksonville Center for Clinical Research

Jacksonville, Florida, 32216, United States

Location

Sunrise Medical Research

Lauderdale Lakes, Florida, 33319, United States

Location

San Marcus Research Clinic, Inc.

Miami, Florida, 33015, United States

Location

Millenium Clinical Research, Inc.

Miami, Florida, 33125, United States

Location

Medcare Research

Miami, Florida, 33165, United States

Location

AR Developoment Solutions

Miami Lakes, Florida, 33014, United States

Location

Soma Medical Research

West Palm Beach, Florida, 33406, United States

Location

Evanston Premier Clinical Research

Evanston, Illinois, 60201, United States

Location

Midwest Institute for Clinical Research, Inc.

Indianapolis, Indiana, 46260, United States

Location

Richard Lochamy, M.D.

Junction City, Kansas, 66441, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

L-MARC Research Center

Louisville, Kentucky, 40213, United States

Location

Clinical Trials Management, LLC

Covington, Louisiana, 70433, United States

Location

Clinical Trials Management, LLC

Metairie, Louisiana, 70006, United States

Location

University of Maryland Medical Center

Baltimore, Maryland, 21201, United States

Location

University of Michigan Health Systems

Ann Arbor, Michigan, 48105, United States

Location

Elite Clinical Research

Jackson, Mississippi, 39202, United States

Location

Clinical Research of South Nevada

Las Vegas, Nevada, 89121, United States

Location

CHEAR Center, LLC

The Bronx, New York, 10455, United States

Location

Advantage Clinical Trials

The Bronx, New York, 10468, United States

Location

Eastern Carolina Medical Clinic

Benson, North Carolina, 27504, United States

Location

Physicians East, NA

Farmville, North Carolina, 27828, United States

Location

Physicians East, NA

Greenville, North Carolina, 27834, United States

Location

Cary Medical Clinic

Morrisville, North Carolina, 27560, United States

Location

Optimed Research

Columbus, Ohio, 43235, United States

Location

PriMed Clinical Research

Dayton, Ohio, 45419, United States

Location

Green and Seidner Family Practice Associates

Lansdale, Pennsylvania, 19446, United States

Location

BTC of Lincoln

Lincoln, Rhode Island, 02865, United States

Location

Carolinas Research Partners, LLC

Rock Hill, South Carolina, 29732, United States

Location

Airline Complete Healthcare

Houston, Texas, 77091, United States

Location

Sante Clinical Research

Kerrville, Texas, 78028, United States

Location

FMC Science

Lampasas, Texas, 76550, United States

Location

Clinical Investigations of Texas

Plano, Texas, 75075, United States

Location

Sun Research Institute

San Antonio, Texas, 78215, United States

Location

Clinical Investigation Specialists

Kenosha, Wisconsin, 53142, United States

Location

Ecogene-21

Chicoutimi, Quebec, G7H 7K9, Canada

Location

MeSH Terms

Conditions

Hypertriglyceridemia

Interventions

gemcabene

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Vice President Clinical Operations
• Organization: NeuroBo Pharmaceuticals

info@neurobopharma.com

+1 (857) 702-9600

Study Officials

• Lee Golden, MD

  NeuroBo Pharmaceuticals Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2016
• First Posted: October 25, 2016
• Study Start: December 1, 2016
• Primary Completion: January 11, 2018
• Study Completion: May 9, 2018
• Last Updated: June 25, 2020
• Results First Posted: June 25, 2020

Record last verified: 2020-06

Locations

US (48); CA (1)