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    Summary
    EudraCT Number:2015-005756-10
    Sponsor's Protocol Code Number:GEM-301
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-03-08
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedDenmark - DHMA
    A.2EudraCT number2015-005756-10
    A.3Full title of the trial
    A Phase 2 Randomized, Double-Blind, Dose-Finding Study to Assess
    the Efficacy, Safety, and Tolerability of Gemcabene in Patients with
    Hypercholesterolemia on a High-Intensity Stable Statin Therapy
    (ROYAL-1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A randomized phase 2 study to determine the dose, and assess
    the efficacy, safety, and tolerability of Gemcabene in Patients with
    Hypercholesterolemia.
    A.3.2Name or abbreviated title of the trial where available
    ROYAL-1
    A.4.1Sponsor's protocol code numberGEM-301
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGemphire Therapeutics Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGemphire Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace, Inc.
    B.5.2Functional name of contact pointMedpace Regulatory Submissions
    B.5.3 Address:
    B.5.3.1Street AddressGmunder Strasse 53
    B.5.3.2Town/ cityMunich
    B.5.3.3Post code81379
    B.5.3.4CountryGermany
    B.5.4Telephone number4989895 57 180
    B.5.5Fax number4989895 571 81 00
    B.5.6E-mailregsubmissions@medpace.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namegemcabene
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNgemcabene calcium
    D.3.9.1CAS number 209789-08-2
    D.3.9.2Current sponsor codeCI-1027 Calcium
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hypercholesterolaemia
    E.1.1.1Medical condition in easily understood language
    High blood cholesterol level
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10020603
    E.1.2Term Hypercholesterolaemia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the efficacy, safety, and tolerability of multiple doses of gemcabene compared to placebo in patients with hypercholesterolemia on a high-intensity stable statin therapy with or without ezetimibe.
    E.2.2Secondary objectives of the trial
    To confirm the appropriate dose for use in Phase 3 registration studies as assessed by efficacy and safety data.
    To further evaluate the efficacy of gemcabene as assessed by measurements of lipid and apolipoprotein parameters, hsCRP, and fibrinogen.
    The exploratory objective of this study is to evaluate the effects of gemcabene on serum PCSK9 levels.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of written and signed informed consent (by patient or legal guardian) prior to any study-specific procedure;
    2. Male or female great than or equal to 18 years of age at time of consent;
    3. Currently on a stable, low-fat, low-cholesterol diet in combination with atorvastatin (40 mg or 80 mg QD) or rosuvastatin (20 mg or 40 mg QD) with or without ezetimibe (10 mg QD) for at least 4 weeks prior to the Screening Visit;
    4. Fasting LDL-C value great than 100 mg/dL at the Screening Visit;
    5. Physical examination, including vital signs, that is within normal limits or clinically acceptable to the Investigator;
    6. Weight great than or equal to 50 kg;
    7. Female patients must not be pregnant or lactating. Women of child-bearing potential must have
    a negative serum pregnancy test at the Screening Visit and negative urine dipstick on 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 greater than or equal to 1 year in the absence of other biological or physiological causes) are not considered to be of child-bearing potential; and
    8. 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.
    E.4Principal exclusion criteria
    1. Abnormal liver function test at the Pre-Screening Visit or the Screening Visit (aspartate aminotransferase or alanine aminotransferase greater than 2 × the upper limit of normal [ULN], total bilirubin greater than 1.5 × ULN, or alkaline phosphatase greater than 2 × ULN based on appropriate age and gender normal values). Patients with bilirubin greater than 1.5 × ULN and 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 [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;
    4. Triglyceride value greater than 400 mg/dL at the Pre-Screening Visit or the Screening Visit;
    5. Moderate to severe renal insufficiency defined as an estimated GFR less than 30 mL/min/1.73m2 (calculated using The Chronic Kidney Disease Epidemiology Collaboration equation) at the Pre-Screening Visit or the 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 ratio testing;
    7. Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by thyroid-stimulating hormone (TSH) below the lower limit of normal or greater than 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;
    8. Type 1 diabetes mellitus or uncontrolled type 2 diabetes mellitus (hemoglobin A1c [HbA1c] value greater than 8% based on results from the Pre-Screening Visit or the Screening Visit), or any diabetic patient taking insulin and/or thiazolidinediones;
    9. New York Heart Association Class III or IV heart failure (see Appendix C);
    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. Patients 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 greater than 180 mmHg or diastolic blood pressure greater than 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. Use 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;
    17. History of drug or alcohol abuse within the past year or inability to comply with protocol requirements, including subject restrictions (see Section 5.6.3);
    18. Previously treated with gemcabene;
    19. Participation in another clinical study of an investigational agent or device concurrently or within 1 month prior to 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; or
    20. Any other finding which, in the opinion of the Investigator, would compromise the patient’s safety or participation in the study.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy analysis is the percent change in LDL-C from baseline to Day 28, Day 56,
    and Day 84.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 28, Day 56, and Day 84
    E.5.2Secondary end point(s)
    The change in LDL-C from baseline;
    The change and percent change in LDL-C from baseline to the average of Day 56 and Day 84 measurements;
    The change and percent change in lipid parameters (non-high-density lipoprotein cholesterol [non-HDL-C], total cholesterol [TC], TG, high-density lipoprotein cholesterol [HDL-C], and very low-density lipoprotein cholesterol [VLDL-C]) from baseline to Day 28, Day 56, and Day 84;
    The number (%) of patients achieving LDL-C reduction of greater than or equal to 10% and greater than or equal to 15% at Day 28, Day 56, and Day 84;
    The number (%) of patients achieving an LDL-C value less than 100 mg/dL at Day 28, Day 56, and Day 84;
    The change and percent change in, apolipoprotein (Apo) B, ApoA-I, ApoA-II, ApoC-II, ApoC-III, ApoE, and lipoprotein(a) (Lp[a]) from baseline to Day 84; and
    The change and percent change in hsCRP and fibrinogen from baseline to Day 84.
    The exploratory analyses are the change and percent change in serum PCSK9 from baseline to Day 84.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 28, Day 56, and Day 84
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Denmark
    Israel
    Netherlands
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days12
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days12
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 32
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state21
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 34
    F.4.2.2In the whole clinical trial 212
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects will be returned to the standard therapy for the treatment of hypercholesterolemia once study participation has been ended.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-04-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2016-07-21
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