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    Summary
    EudraCT Number:2016-000906-12
    Sponsor's Protocol Code Number:AQX-1125-301
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-07-19
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-000906-12
    A.3Full title of the trial
    The LEADERSHIP 301 Trial: A 12-Week, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, 3 Arm, Parallel-Group, Phase 3 Trial to Evaluate the Efficacy and Safety of 2 Doses of AQX-1125 Targeting the SHIP1 Pathway in Subjects with Interstitial Cystitis/Bladder Pain Syndrome Followed by a 40-Week Extension Period
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A randomized study comparing Placebo and AQX-1125 in the treatment of subjects with Interstitial Cystitis/Bladder Pain Syndrome
    A.3.2Name or abbreviated title of the trial where available
    The LEADERSHIP 301 Trial
    A.4.1Sponsor's protocol code numberAQX-1125-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02858453
    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 SponsorAquinox Pharmaceuticals (Canada) Inc.
    B.1.3.4CountryCanada
    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 supportAquinox Pharmaceuticals (Canada) Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAquinox Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address1150 Bayhill Drive, Suite 200
    B.5.3.2Town/ citySan Bruno
    B.5.3.3Post codeCA 94066
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1650295 1423
    B.5.5Fax number+1650634 0830
    B.5.6E-mailclinops@aqxpharma.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.3.2Product code AQX-1125
    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 INNAQX-1125
    D.3.9.1CAS number 782487-29-0
    D.3.9.2Current sponsor codeAQX-1125
    D.3.9.3Other descriptive nameAQX-1125
    D.3.9.4EV Substance CodeSUB182776
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboFilm-coated tablet
    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
    Interstitial Cystitis/Bladder Pain Syndrome
    E.1.1.1Medical condition in easily understood language
    Interstitial Cystitis/Bladder Pain Syndrome
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level LLT
    E.1.2Classification code 10071166
    E.1.2Term Bladder pain syndrome
    E.1.2System Organ Class 100000004857
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level LLT
    E.1.2Classification code 10008927
    E.1.2Term Chronic interstitial cystitis
    E.1.2System Organ Class 100000004857
    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 evaluate the effect of 12 weeks of treatment with 2 different doses of oral AQX-1125 (100 mg or 200 mg) administered once daily compared to placebo on the change from Baseline (Visit 2) to Week 12 (Visit 4) in maximum daily bladder pain in subjects with IC/BPS using a standardized 11-point NRS pain score recorded daily by e-diary.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate:
    • The effect of 12 weeks of treatment with 2 different doses of oral AQX-1125 (100 mg or 200 mg) administered once daily compared to placebo on the change from Baseline (Visit 2) to Week 12 (Visit 4) for each of the following:
    o Urinary voiding frequency over a 24-hour period.
    o ICSI.
    o BPIC-SS.
    • Overall response to treatment for AQX-1125 100 mg or 200 mg compared to placebo as measured by the subject’s Global Response Assessment (GRA) at Week 12.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    For inclusion in to the screening period subjects must meet the following criteria:
    1. Provide written informed consent and the willingness and ability to comply with all aspects of the study requirements.
    2. Males/females, ≥18 and ≤80 years of age at Screening Visit 1.
    3. Subjects who have consistently had symptoms of bladder pain in addition to urinary urgency and/or urinary frequency for more than 6 months (to ensure a properly established diagnosis).
    4. Have had the clinical diagnosis, or history consistent with the diagnosis, of IC/BPS for >3 months but ≤20 years (to ensure a properly established diagnosis).
    5. BPIC-SS minimum score as per the study protocol.
    6. ICSI minimum score as per the study protocol.
    7. Pelvic floor pain maximum score as per the study protocol on the 11-point NRS pain scale following a pelvic pain assessment (to discriminate between bladder pain and perineal/pelvic floor pain masquerading as bladder pain).
    8. Must be capable of voiding independently (to allow completion of voiding diary over a 24 hour period).
    9. Subjects must fulfil at least one of the following criteria:
    • Males/females surgically sterile for a minimum of 6 months; or
    o Females: Post-menopausal for a minimum of 1 year; or
    o If of child bearing potential, must have a negative pregnancy test and agree to avoid pregnancy and use a highly effective method of contraception with one additional barrier method of contraception from Screening Visit 1 to the final Follow-up Visit of the study (or until at least 28 days after the last dose of study drug has been taken).
    o Acceptable methods of highly effective contraception include non-hormonal intrauterine device, intrauterine hormone-releasing system or hormonal contraception (patch, injectable or implantable). Acceptable barrier methods include male condom, diaphragm, cap or sponge, or vasectomy of sole sexual partner.
    o If using oral hormonal contraception, the barrier method used must include spermicide.
    o True abstinence can be used as a method of contraception, when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception.
    o Males: Must use a condom for sexual intercourse from Screening Visit 1 until at least 90 days after last dose of study drug has been taken, unless they have been surgically sterilized (vasectomy).
    10. Women of child bearing potential must have a negative pregnancy test at Screening Visit 1, Baseline (Visit 2) and throughout the study.
    11. Females are non-lactating (Screening Visit 1, Baseline [Visit 2] and throughout the study).

    For inclusion into TP1, subjects must meet the following criteria at Baseline (Visit 2):
    12. Have minimum average daily pain score as per the study protocol on the 11-point NRS pain scale (mean of the average daily pain score recorded at each of the 7 days prior to Baseline [Visit 2]).
    13. Minimum number of urinary voids as per the study protocol in a 24-hour period recorded within 3 days (72 hours) prior to Baseline (Visit 2).
    14. Have undergone a cystoscopy within the last 36 months (inclusive) prior to Baseline (Visit 2). For cystoscopies performed prior to Screening Visit 1, results of that cystoscopy must be available and include presence or absence of Hunner Lesion and additional pathology.
    • If the cystoscopy was performed for non-therapeutic purposes, it must have been performed at least 14 days prior to Screening Visit 1.
    • If the cystoscopy involved therapeutic hydrodistension, it must have been performed at least 3 months prior to Baseline (Visit 2). The results of that cystoscopy must be available and the information, particularly the presence or absence of Hunner Lesion will be collected; or
    • If no cystoscopy has been performed prior to Screening Visit 1, the results are unavailable or do not meet the requirements of the protocol, the subject will have a cystoscopy (without hydrodistension) at Screening Visit 1a, within 14 days of Screening Visit 1 (with Baseline [Visit 2] occurring a minimum of 14 days and a maximum of 28 days after the screening cystoscopy at Screening Visit 1a).
    E.4Principal exclusion criteria
    Subjects meeting any of the following criteria at Screening Visit 1 will not be eligible for study participation. However, subjects fulfilling criterion 2 will be eligible for rescreening:
    1. Catastrophizing pain score over maximum score allowed per the study protocol as determined by the Pain Catastrophizing Scale (PCS).
    2. Have had a urinary tract infection (UTI) including bacterial cystitis within the past 30 days (inclusive) or presence on laboratory C&S at Screening Visit 1. Subjects with current infection may be treated according to standard of care and rescreened at least 10 days after resolution of infection (and have a repeat urine C&S that was documented as clear).
    3. Microscopic hematuria that has not been adequately evaluated per local standard of care.
    4. History of chronic substance abuse, dependency or abuse of opiates, or other narcotics within the last 2 years.
    5. Currently receiving any of the following prohibited medications or procedures:
    • Taken antihistamine or NSAID unless on a stable dose for ≥30 days prior to Screening Visit 1.
    • Taken any long-acting opiates within 2 weeks prior to Baseline (Visit 2) and throughout the study, or more than 10 short-acting opiates/month. If (short-acting) opiate analgesics are taken during the screening period they should be limited to a maximum of 2 days per week and not within 3 days prior to randomization.
    • Oral steroid or cyclosporine therapy within 30 days prior to Screening Visit 1 and throughout the study.
    • Had treatment with intravesical therapy within 60 days prior to Screening Visit 1.
    • Bladder hydrodistension and/or fulguration within 3 months prior to Screening Visit 1 and throughout the study.
    • Have taken any investigational drug within 90 days prior to Screening Visit 1 or have had previous exposure to AQX-1125.
    6. History of previous procedure(s) (augmentation cystoplasty, cystectomy, cystolysis, botulinum toxin or bladder catheterization) that has significantly affected bladder function.
    7. History of cyclophosphamide or chemical cystitis, urinary tuberculosis or radiation cystitis.
    8. Females: History of bladder tumors or uterine, cervical, vaginal or urethral cancer.
    9. Males: History of prostate surgery (TURP, TURT, TUIP TUNA etc.), a history of prostate cancer, or currently being treated for chronic bacterial prostatitis.
    10. Have any other condition/disease which, in the opinion of the Investigator, could compromise subject safety or interfere with the subject’s participation in the study or in the evaluation of the study results. In case of any doubt, the Investigator shall consult the Medical Monitor.
    11. Major surgery within 3 months prior to Screening Visit 1.
    12. Known intolerance to micro-crystalline cellulose (Avicel® PH-102), mannitol or other ingredient of AQX-1125 tablets.
    E.5 End points
    E.5.1Primary end point(s)
    The change from Baseline (Visit 2) at Week 12 (Visit 4) for AQX-1125 100 mg or 200 mg compared to placebo in the maximum daily bladder pain score based on a standardized 11 point NRS recorded by e-diary as measured by the mean of the maximum scores recorded once daily for a minimum of 5 of the 7 days prior to each visit.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Once daily for a minimum of 5 of the 7 days prior to each of Baseline (Visit 2) , Week 6 (Visit 3) and Week 12 (Visit 4).
    E.5.2Secondary end point(s)
    The key secondary endpoints are:
    • The change from Baseline (Visit 2) at Week 12 (Visit 4) for AQX-1125 100 mg or 200 mg compared to placebo in the following:
    o Voiding frequency measured over a 24-hour period, within a 3 day (72 hours) window before Baseline (Visit 2) and again before Week 12 (Visit 4).
    o ICSI.
    o BPIC-SS.
    • Overall response to treatment for AQX-1125 100 mg or 200 mg compared to placebo as measured by the subject’s GRA at Week 12.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Voiding frequency - measured over a 24-hour period, within a 3 day (72 hours) window before Baseline (Visit 2) and again before Week 12 (Visit 4).
    ICSI/BPIC-SS - Baseline (Visit 2), Week 6 (Visit 3) and Week 12 (Visit 4)
    GRA - Week 6 (Visit 3) and Week 12 (Visit 4)
    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 Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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 trial3
    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 EEA62
    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
    Belgium
    Canada
    Czech Republic
    Denmark
    Germany
    Hungary
    Latvia
    Netherlands
    Poland
    Romania
    Spain
    United Kingdom
    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
    LSLV at the end of the 40-week extension period
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 450
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 150
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 225
    F.4.2.2In the whole clinical trial 600
    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)
    None
    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 Decision2017-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-06-27
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