A 12-Week, Phase 2 Study of Gemcabene in Hypercholesterolemia Patients on Stable Moderate and High-Intensity Statins (ROYAL-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02634151
Recruitment Status : Completed
First Posted : December 17, 2015
Last Update Posted : August 25, 2017
Information provided by (Responsible Party):
Gemphire Therapeutics, Inc.

Brief Summary:
The purpose of this study is to assess the efficacy, safety, and tolerability of multiple doses of gemcabene 600 mg QD compared to placebo in patients with hypercholesterolemia not adequately controlled on high-intensity or moderate-intensity stable statin therapy. Patients with HeFH, ASCVD, or otherwise uncontrolled, may be included with baseline LDL-C value ≥ 100 mg/dL. Subjects will be randomized 1:1 to gemcabene 600 mg QD or placebo.

Condition or disease Intervention/treatment Phase
Hypercholesteremia Drug: Gemcabene 600 mg Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 105 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A 12-Week, Phase 2 Randomized, Placebo-Controlled, Double-Blind Study to Assess the Efficacy, Safety and Tolerability of Gemcabene in Subjects With Hypercholesterolemia Not Adequately Controlled on High-Intensity or Moderate-Intensity Stable Statin Therapy
Study Start Date : November 2016
Actual Primary Completion Date : June 2017
Actual Study Completion Date : August 2017

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Placebo Comparator: Placebo
Placebo treatment on stable background statin therapy
Drug: Placebo
Placebo, 3 tabs QD, 84 days

Experimental: Gemcabene 600 mg QD
Gemcabene treatment on stable background statin therapy
Drug: Gemcabene 600 mg
2-300 mg tablets, 1 placebo tablet, QD, 84 days

Primary Outcome Measures :
  1. LDL-C [ Time Frame: Baseline and 84 days (12 weeks) ]
    % change from baseline

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  1. Provision of written and signed informed consent (by subject or legal guardian) prior to any study-specific procedures;
  2. Male or female (neither pregnant or lactating) ≥ 18 years of age at the time of consent;
  3. Currently on a stable, low-fat, low-cholesterol diet in combination with allowed statin doses as described in Table 1, with or without ezetemibe 10 mg QD for at least 12 weeks prior to the Screening Visit;
  4. Fasting LDL-C value ≥ 100 mg/dL (2.59 mmol/L) at the Screening Visit;
  5. Physical examination, including vital signs, that is within normal limits or clinically acceptable to the Investigator;
  6. Weight ≥ 50 kg; with a body mass index (BMI) ≤ 45 kg/m2
  7. Subjects with Type 2 diabetes who take anti-hyperglycemic agents must be on a stable regimen for at least 3 months, with no planned changes in medications for the study duration.

Exclusion Criteria

  1. Abnormal liver function test at the Pre-Screening or Screening Visit (AST or ALT) > 2x ULN (upper limit of normal), total bilirubin > 1.5x ULN, or alkaline phosphate > 2x ULN based on appropriate age and gender normal values. Subjects with bilirubin > 1.5x ULN and a history of Gilbert's syndrome may be included; relfexive direct bilirubin testing will be used to confirm Gilbert's syndrome;
  2. Moderate (Grade B) or severe (Grade C) chronic hepatic impairment according to the Child-Pugh classification;
  3. Active liver disease (e.g. cirrhosis, alcoholic liver disease, hepatitis B, hepatitis C, automimmune hepatitis, liver failure, liver cancer), history of liver transplant, known diagnosis of HIV or AIDS;
  4. Triglyceride value ≥ 500 mg/dL at the Pre-Screening Visit or the Screening Visit;
  5. Moderate to severe renal insufficiency define as an estimated GFR < 60mL/min/1.73m (calculated using the Chronic Kidney Disease Epidemiology Collaboration equation) at the Pre-Screening Visit or Screening Visit;
  6. Abnormal urinalysis (proteinuria greater than trace or any male or non-menstruating female with greater than trace hematuria) confirmed by reflexive urine protein:creatinine ration testing;
  7. Uncontrolled thyroid disease; hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) below the lower limit of normal or > 1.5x 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 Screening;
  8. Type 1 diabetes mellitus or uncontrolled type 2 diabetes mellitus (hemoglobin A1c value > 8.5% based on results from the Pre-Screening or Screening Visit, or taking a thiazolidinedione (i.e. pioglitazone or rosiglitazone);
  9. New York Heart Association Class III or IV heart failure;
  10. 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. Subjects with adequately treated stable angina, per Investigator assessment, may be included;
  11. Uncontrolled cardiac arrhythmia or prolonged QT on the Screening Visit or 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;
  12. Uncontrolled hypertension, defined as sitting systolic blood pressure > 180 mmHg or diastolic blood pressure > 110 mmHg, and confirmed by repeat measurement;
  13. Currently receiving cancer treatments or, in the Investigator's opinion, at risk of relapse for recent cancer;
  14. Inadequate wash-out of a PCSK9 inhibitor (8 weeks prior to the Screening Visit), a fibrate lipid-regulating agent (6 weeks prior to the Screening Visit), niacins (4 weeks prior to the Screening Visit), or other lipid-regulating therapies such as bile acid sequestrants (4 weeks prior to the Screening Visit);
  15. Hypersensitivity to or a history of significant adverse reactions to any fibrate lipid-regulating agent;
  16. Use of any excluded medications or supplements (e.g. potent cytochrome P450 [CYP] 3A4 inhibitors as described in Appendix D;
  17. History of drug or alcohol abuse within the past year or inabilty to comply with protocol requirements, including subjects restrictions (see Section 5.6.3);
  18. Previously treated with gemcabene (CI-1027), participation in another clinical study of an investigational agent or device concurrently or within 1 month prior the Screening Visit, or use of an investigational agent within 1 month or 5 half-lives (if known), whichever is longer, prior to the Screening Visit;
  19. Any other finding which, in the opinion of the Investigator, would compromise the subject's safety or participation in the study.

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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02634151

United States, Alabama
Central Research Associates, Inc.
Birmingham, Alabama, United States, 35205
United States, California
Westside Medical Associates of Los Angeles
Beverly Hills, California, United States, 90211
National Research Institute
Huntington Park, California, United States, 90255
National Research Institute
Los Angeles, California, United States, 90057
United States, Florida
Atlantic Clinical Research Collaborative- Cardiology
Atlantis, Florida, United States, 33462
Excel Medical Clinical Trials
Boca Raton, Florida, United States, 33434
Jacksonville Center for Clinical Research
Jacksonville, Florida, United States, 32216
Health Awareness, Inc.
Jupiter, Florida, United States, 33458
Meridien Research, Inc.
Maitland, Florida, United States, 32751
Progressive Medical Research
Port Orange, Florida, United States, 32127
Varkey Medical
Tampa, Florida, United States, 33626
United States, Illinois
Evanston Premier Healthcare Research, LLC
Evanston, Illinois, United States, 60201
United States, Indiana
Midwest Institute for Clinical Research
Indianapolis, Indiana, United States, 46260
United States, Kentucky
Louisville, Kentucky, United States, 40213
United States, New York
Mid-Hudson Medical Research
New Windsor, New York, United States, 12553
Rochester Clinical Research, Inc.
Rochester, New York, United States, 14609
United States, Ohio
Sterling Research Group, Ltd.
Cincinnati, Ohio, United States, 45219
Sentral Clinical Research Services
Cincinnati, Ohio, United States, 45236
Sterling Research Group, Ltd.
Cincinnati, Ohio, United States, 45246
United States, Pennsylvania
Green and Seidner Family Practice Associates
Lansdale, Pennsylvania, United States, 19446
United States, Texas
Associates in Medicine
Houston, Texas, United States, 77027
Diagnostics Research Grup
San Antonio, Texas, United States, 78229
Sugar Lakes Family Practice
Sugar Land, Texas, United States, 77479
United States, Virginia
National Research Institute
Richmond, Virginia, United States
Sponsors and Collaborators
Gemphire Therapeutics, Inc.

Responsible Party: Gemphire Therapeutics, Inc. Identifier: NCT02634151     History of Changes
Other Study ID Numbers: GEM-301
First Posted: December 17, 2015    Key Record Dates
Last Update Posted: August 25, 2017
Last Verified: August 2017

Keywords provided by Gemphire Therapeutics, Inc.:
Lipid Regulator

Additional relevant MeSH terms:
Lipid Metabolism Disorders
Metabolic Diseases
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Enzyme Inhibitors
Lipid Regulating Agents