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    Summary
    EudraCT Number:2015-003364-36
    Sponsor's Protocol Code Number:GED-0301-UC-002
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-01-28
    Trial results View 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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2015-003364-36
    A.3Full title of the trial
    A Phase 2, Open-label, Multicenter Study to Explore the Efficacy and Safety of Mongersen (GED-0301) in Subjects with Active Ulcerative Colitis.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study using a new drug (GED-0301) to investigate if the new drug works and is safe in subjects suffering ulcerative colitis.
    A.4.1Sponsor's protocol code numberGED-0301-UC-002
    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 SponsorCelgene Corporation
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1888 260 1599
    B.5.5Fax number+1913 266 0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 nameMongersen
    D.3.2Product code GED-0301
    D.3.4Pharmaceutical form Film-coated 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 INNMongersen
    D.3.9.1CAS number 1443994-86-6
    D.3.9.2Current sponsor codeGED-0301
    D.3.9.3Other descriptive nameGED-0301
    D.3.9.4EV Substance CodeSUB30963
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Ulcerative Colitis.
    E.1.1.1Medical condition in easily understood language
    A type of inflammatory bowel disease.
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10045365
    E.1.2Term Ulcerative colitis
    E.1.2System Organ Class 100000004856
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To explore the effect of GED-0301 on clinical activity, as measured by the MMS in subjects with active UC.
    E.2.2Secondary objectives of the trial
    1. To explore the effect of GED-0301 on endoscopic outcome, as measured by the Mayo endoscopic subscore, in subjects with active UC.
    2. To evaluate the safety and tolerability of GED-0301 in subjects with active UC.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is ≥ 18 years of age at the time of signing the ICF.
    2. Subject is able to understand and voluntarily sign an ICF prior to conducting any studyrelated assessments/procedures.
    3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
    4. Subject must have a diagnosis of UC with a duration of at least 3 months prior to screening.
    5. Subject must have moderate to severe UC, defined as MMS ≥ 4 to ≤ 9 with RBS ≥ 1 at screening.
    6. Subject must have a Mayo endoscopic subscore ≥ 2 at screening.
    7. Subject must have failed or experienced intolerance to at least one of the following: aminosalicylates; budesonide; systemic corticosteroids; immunosuppressants (eg, 6-mercaptopurine [6-MP], or azathioprine [AZA], or TNF-α blockers (eg, infliximab, adalimumab, or golimumab).
    8. Subject must meet the following laboratory criteria:
    a. White blood cell count ≥ 3000/mm3 (≥ 3.0 X 10 9/L)
    b. Platelet count ≥ 100,000/mm3 (≥ 100 X 10 9/L)
    c. Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L)
    d. Aspartate transaminase (AST/serum glutamic oxaloacetic transaminase [SGOT])
    and alanine transaminase (ALT/serum pyruvic transaminase [SGPT]) ≤ 2.5 X upper limit of normal (ULN)
    e. Total bilirubin ≤ 2 mg/dL (≤ 34 μmol/L) unless there is a confirmed diagnosis of Gilbert's disease
    f. Hemoglobin ≥ 9 g/dL (≥ 5.6 mmol/L)
    g. Activated partial thromboplastin time (APTT) ≤ 1.5 X ULN
    9. Females of childbearing potential (FCBP1) must have a negative pregnancy test at the Screening and Baseline Visits. While on IP and for at least 28 days after taking the last dose of IP, FCBP who engage in activity in which conception is possible must use one of the approved contraceptive options described below:
    Option 1: Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner’s vasectomy;
    OR
    Option 2: Male or female condom PLUS 1 additional barrier method: (a) diaphragm
    with spermicide; (b) cervical cap with spermicide; or (c) contraceptive sponge with spermicide.
    10. Male subjects (including those who have had a vasectomy) when engaging in sexual activity with females who are able to become pregnant must use barrier contraception (male latex condom or nonlatex condom NOT made out of natural [animal] membrane [for example, polyurethane]) while on IP and for at least 28 days after the last dose.
    E.4Principal exclusion criteria
    1. Subject has a diagnosis of CD, indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis.
    2. Subject has ulcerative colitis restricted to distal 15 cm or less (eg, ulcerative proctitis).
    3. Subject had surgery as a treatment for UC or who, in the opinion of the Investigator, is likely to require surgery for UC during the study.
    4. Subject has clinical signs suggestive of fulminant colitis or toxic megacolon.
    5. Subject is stool positive for any enteric pathogen or Clostridium difficile (C. difficile) toxin at screening.
    6. Subject has history of colorectal cancer or colorectal dysplasia.
    7. Prior treatment with more than 2 TNF-α blockers (eg, infliximab, adalimumab, or golimumab).
    8. Prior treatment with any integrin antagonists (eg, natalizumab or vedolizumab).
    9. Use of TNF-α blockers within 8 weeks of the screening.
    10. Subject had prior treatment with mycophenolic acid, tacrolimus, sirolimus, cyclosporine, thalidomide or apheresis (eg, Adacolumn®) for the treatment of UC. In addition, prior use of any of these treatment modalities for an indication other than UC within 8 weeks of screening is also excluded.
    11. Subject has received intravenous (IV) corticosteroids within 2 weeks of screening.
    12. Subject has received topical treatment with 5-ASA or corticosteroid enemas or suppositories within 2 weeks of screening.
    13. Subject has received total parenteral nutrition (TPN) within 4 weeks of screening.
    14. Subject has a history of any clinically significant neurological, renal, hepatic, gastrointestinal, pulmonary, metabolic, cardiovascular, psychiatric, endocrine, hematological disorder or disease, or any other medical condition that, in the investigator's opinion, would prevent the subject from participation in the study.
    15. Subject has any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she was to participate in the study or confounds the ability to interpret data from the study.
    16. Subject is pregnant or breastfeeding.
    17. Subject has a history of any of the following cardiac conditions within 6 months of screening: myocardial infarction, acute coronary syndrome, unstable angina, new onset atrial fibrillation, new onset atrial flutter, second- or third-degree atrioventricular block, ventricular fibrillation, ventricular tachycardia, heart failure, cardiac surgery, interventional cardiac catheterization (with or without a stent placement), interventional electrophysiology procedure, or presence of implanted defibrillator.
    18. Subject has a known active current or history of medically important recurrent bacterial, viral, fungal, mycobacterial or other infections (including but not limited to tuberculosis and atypical mycobacterial disease and Herpes zoster), human immunodeficiency virus (HIV), or any major episode of infection requiring hospitalization or treatment with IV or oral antibiotics within 4 weeks of screening.
    19. Subject has a history of congenital or acquired immunodeficiency (eg, common variable immunodeficiency disease).
    20. Subject has a history of malignancy, except for:
    a. Treated (ie, cured) basal cell or squamous cell in situ skin carcinomas
    b. Treated (ie, cured) cervical intraepithelial neoplasia or carcinoma in situ of the cervix with no evidence of recurrence within the previous 5 years
    21. Subject has received investigational drug or device within 1 month of screening.
    22. Subject has a history of alcohol, drug, or chemical abuse within the 6 months prior to screening.
    23. Subject has a known hypersensitivity to oligonucleotides or any ingredient in the IP.
    24. Subject has prior treatment with GED-0301 or participated in a clinical study involving GED-0301.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy:
    The proportion of subjects achieving clinical remission in the MMS, defined as a MMS of ≤ 2, with no individual subscore > 1.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 8.
    E.5.2Secondary end point(s)
    Efficacy:
    1. The proportion of subjects achieving a MMS of ≤ 2, with rectal bleeding subscore of 0 and stool frequency subscore and Mayo endoscopic subscore ≤ 1.
    2. The proportion of subjects achieving a Mayo endoscopic subscore ≤ 1.
    3. The proportion of subjects achieving a Mayo endoscopic subscore by individual segment (rectum, sigmoid, descending colon, transverse colon, ascending colon/cecum) ≤ 1.
    4. The proportion of subjects achieving clinical response in the MMS, defined as a decrease from baseline of at least 2 points and at least 25%, along with a reduction in the RBS of at least 1 point or an absolute RBS ≤ 1.
    5. The proportion of subjects achieving endoscopic response, defined as a decrease from baseline of at least 1 point in the Mayo endoscopic subscore.
    6. The proportion of subjects achieving clinical remission in the TMS, defined as a TMS of ≤ 2, with no individual subscore > 1.
    7. The proportion of subjects achieving clinical response in the TMS, defined as a decrease from baseline in the TMS of at least 3 points and at least 30%, along with a reduction in the RBS of at least 1 point or an absolute RBS of ≤ 1.
    Safety:
    The evaluation of safety and tolerability of GED-0301, assessed by the type, frequency and severity of adverse events, and its relationship to IP, discontinuation due to adverse events, and clinically significant changes in vital signs, ECGs, and/or laboratory findings.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy: Week 8.
    Safety: Through Week 52.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Biomarkers.
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Bulgaria
    Canada
    Hungary
    Poland
    Slovakia
    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
    Either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, whichever is the later date.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 40
    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)
    Best standard of care at investigator's discretion.
    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 Decision2016-02-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-08-08
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