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

June 3, 2020 updated by: NeuroBo Pharmaceuticals Inc.

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

The purpose of this study was 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 were randomized 1:1 to gemcabene 600 mg once daily (QD) or placebo.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
105

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  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 ezetimibe 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; reflexive 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, autoimmune 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 inability 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Participants on stable statin therapy received matching placebo orally, once daily for 12 weeks.
Drug: Placebo
Three placebo tablets administered orally once daily for 12 weeks.
Experimental: Gemcabene 600 mg
Participants on stable statin therapy received 600 milligrams (mg) of Gemcabene orally, once daily for 12 weeks.
Drug: Gemcabene
Two 300 mg tablets and 1 placebo tablet administered orally, once daily for 12 weeks.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Percent Change From Baseline in LDL-C at Week 12
Time Frame: Baseline, Week 12
Baseline, Week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in LDL-C by Statin Intensity Stratum
Time Frame: Baseline, Week 12
The intensity of statin therapy was determined based on the statin dose.Participants were categorized as high intensity & moderate intensity based on their statin doses.
Baseline, Week 12
Change From Baseline in LDL-C
Time Frame: Baseline, Weeks 2, 4, 8, 12 and average of weeks 8 and 12
Baseline, Weeks 2, 4, 8, 12 and average of weeks 8 and 12
Percent Change From Baseline in LDL-C
Time Frame: Baseline, average of weeks 8 and 12
Baseline, average of weeks 8 and 12
Percent Change From Baseline in Non-HDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Change From Baseline in Non-HDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Percent Change From Baseline in TC
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Change From Baseline in TC
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Percent Change From Baseline in TG
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Change From Baseline in TG
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Percent Change From Baseline in VLDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Change From Baseline in VLDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Percent Change From Baseline in HDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Change From Baseline in HDL-C
Time Frame: Baseline, Weeks 2, 4, 8 and 12
Baseline, Weeks 2, 4, 8 and 12
Number of Participants Achieving LDL-C Reduction of ≥10%
Time Frame: Weeks 4, 8 and 12
Weeks 4, 8 and 12
Number of Participants Achieving LDL-C Reduction of ≥15%
Time Frame: Weeks 4, 8 and 12
Weeks 4, 8 and 12
Number of Participants Achieving LDL-C Reduction of ≥20%
Time Frame: Weeks 4, 8 and 12
Weeks 4, 8 and 12
Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L)
Time Frame: Weeks 4, 8 and 12
Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein B
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein B
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein A-I
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein A-I
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein A-II
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein A-II
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein C-II
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein C-II
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein C-III
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein C-III
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Apolipoprotein E
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Apolipoprotein E
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in Lipoprotein(a)
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Change From Baseline in Lipoprotein(a)
Time Frame: Baseline, Weeks 4, 8 and 12
Baseline, Weeks 4, 8 and 12
Percent Change From Baseline in High-sensitivity C-reactive Protein
Time Frame: Baseline, Week 12
Baseline, Week 12
Change From Baseline in High-sensitivity C-reactive Protein
Time Frame: Baseline, Week 12
Baseline, Week 12
Percent Change From Baseline in Fibrinogen
Time Frame: Baseline, Week 12
Baseline, Week 12
Change From Baseline in Fibrinogen
Time Frame: Baseline, Week 12
Baseline, Week 12
Percent Change From Baseline in Serum Amyloid A
Time Frame: Baseline, Week 12
Baseline, Week 12
Change From Baseline in Serum Amyloid A
Time Frame: Baseline, Week 12
Baseline, Week 12
Percent Change From Baseline in Adiponectin
Time Frame: Baseline, Week 12
Baseline, Week 12
Change From Baseline in Adiponectin
Time Frame: Baseline, Week 12
Baseline, Week 12
Change From Baseline in Framingham Risk Score
Time Frame: Baseline, Week 12
Framingham Risk Score was an estimate of a participant's 10-year risk of developing cardiovascular disease which was computed using sex-specific algorithms based on total point score (range less than negative 3 [best] to greater than or equal to 14 [worst] for men; less than or equal to negative 2 [best] and greater than or equal to 17 [worst] for women) : which was derived of participant's age, systolic blood pressure , smoking status, TC, HDL-C, treatment for hypertension, and diabetes status. Reported score is a percentage. Change from baseline calculated as mean at week 12 minus mean at baseline. Negative scores indicate less risk of developing cardiovascular disease.
Baseline, Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
NeuroBo Pharmaceuticals Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 11, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 15, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 17, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 25, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 3, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GEM-301

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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