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    Summary
    EudraCT Number:2018-004419-32
    Sponsor's Protocol Code Number:PMS-2018-002
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-03-19
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2018-004419-32
    A.3Full title of the trial
    A Phase IIa, Proof of Concept, Randomized, Double-Blind, Dose-Finding, Cross-Over Study of the Efficacy, Safety and Tolerability of a New Enteric-Coated Cholestyramine Capsule in Adult Short Bowel Syndrome Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial to Test the Efficacy, Safety and Tolerability of Two Dosing Regimens of Cholestyramine Coated Capsules in Adults with Short Bowel Syndrome
    A.4.1Sponsor's protocol code numberPMS-2018-002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04046328
    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 SponsorPharmascience 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 supportPharmascience Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharmascience Inc.
    B.5.2Functional name of contact pointGlobal Specialty Pharma
    B.5.3 Address:
    B.5.3.1Street Address6111 Royalmount Avenue
    B.5.3.2Town/ cityMontreal
    B.5.3.3Post codeH4P 2T4
    B.5.3.4CountryCanada
    B.5.4Telephone number001514865-5022
    B.5.6E-mailstanguay@pharmascience.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 nameEnteric-Coated Cholestyramine (ECC) Capsules
    D.3.2Product code 37180
    D.3.4Pharmaceutical form Modified-release capsule, hard
    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 INNCOLESTYRAMINE
    D.3.9.1CAS number 11041-12-6
    D.3.9.2Current sponsor code1221
    D.3.9.3Other descriptive nameCholestyramine Resin USP, DUOLITE AP143/1083
    D.3.9.4EV Substance CodeSUB01427MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number425
    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 placeboModified-release capsule, hard
    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
    Bile-acid induced diarrhea in adult patients with Short Bowel Syndrome (SBS)
    E.1.1.1Medical condition in easily understood language
    Diarrhea in adults with Short Bowel Syndrome (SBS)
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10049416
    E.1.2Term Short-bowel syndrome
    E.1.2System Organ Class 10017947 - Gastrointestinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10080049
    E.1.2Term Bile acid diarrhea
    E.1.2System Organ Class 10017947 - Gastrointestinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to investigate the clinical efficacy of new ECC capsules and select the most effective dose in adult patients with SBS.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate the safety and tolerability of new ECC capsules in adult patients with SBS, and to evaluate the patients’ experience of related symptoms using a 11-point Visual Analog Scale (VAS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Adult, ambulatory male and female subjects
    2. Provision of signed and dated informed consent form (ICF)
    3. Age ≥ 18 years and ≤ 80 years
    4. Stable SBS of:
    a. Non-surgical origin; OR
    b. Surgical origin where the last surgical ileal resection was performed at least 6 months prior to enrolment
    5. Partial, Home Parenteral Nutrition and/or parenteral fluids are allowed, at a maximum frequency of 6 times a week throughout the trial, as long as the regimen has been stable for at least 2 weeks prior to screening and is expected to remain unchanged during the study
    6. At least 50 % of the colon being intact
    7. Intact duodenum
    8. BMI ≥ 18
    9. Presence of stable chronic diarrhea for at least 3 months prior to enrolment as evidenced by medical history
    10. Presence of stable chronic diarrhea during the 2-week screening diary period before randomization, as evidenced by completion of a screening diary demonstrating:
    a. Mean daily production of at least 3 soft or watery stools (BSFS scores 6 or 7); or
    b. More than 3 bowel movements per day on average with >25% of them being BSFS type 6 or 7
    11. Stated willingness and ability to comply with all study procedures, including daily recording of bowel movements and BSFS in the patient diaries, and availability for the duration of the study
    12. Clinical laboratory values within the laboratory's stated normal range; if not within this range, they must be without clinical significance, as determined by the investigator
    13. Female subjects must meet one of the following criteria:
    a) Participant is of childbearing potential and agrees to use one of the accepted contraceptive regimens from at least 30 days prior to the first study treatment administration through to at least 30 days after the last dose of the study treatment. An acceptable method of contraception includes one of the following:
    a. Abstinence from heterosexual intercourse
    b. Systemic contraceptives (combined birth control pills, injectable/implant/insertable hormonal birth control products, transdermal patch)
    c. Intrauterine device (with or without hormones)
    d. Condom with spermicide
    b) Participants of non-childbearing potential, defined as surgically sterile (i.e. has undergone complete hysterectomy or bilateral oophorectomy) or is in a menopausal state (i.e. at least 1 year without menses prior to the first study drug administration) are eligible
    E.4Principal exclusion criteria
    1. Patients with known or suspected intestinal strictures of clinical relevance as judged by the Investigator
    2. Active inflammatory bowel disease (IBD) or fistula during the screening period as judged by the Investigator
    3. Crohn's disease patients not being in clinical remission for the last 12 weeks prior to randomization
    4. Diarrhea caused by other causes than SBS
    5. Presence of clinically significant steatorrhea, requiring pancreatic enzymes supplementation
    6. Presence of complete biliary obstruction
    7. Presence of active cancer (except resected cutaneous basal or squamous cell carcinoma and except in situ cervical cancer) and/or need to receive chemotherapy or radiotherapy during the study
    8. History of allergic reaction to cholestyramine or any excipient of the investigational drug product or placebo, or packaging components
    9. Females who are lactating at screening
    10. Females who are pregnant according to the pregnancy test at screening or prior to the first study treatment administration
    11. Significant history (at least 3 consecutive months in the year prior to Screening) of drug dependency or alcohol abuse (> 3 units of alcohol per day, intake of excessive alcohol, acute or chronic)
    12. Subjects who took an Investigational Product (IP) in the 30 days prior to the first study drug administration
    13. Any other clinically significant condition that is considered by the principal investigator as being susceptible to put the patient at greater safety risk, influence response to study product, or interfere with study assessments.
    E.5 End points
    E.5.1Primary end point(s)
    Change in the weekly frequency of bowel movements measured between baseline and the second week of treatment
    E.5.1.1Timepoint(s) of evaluation of this end point
    Days 8 to 14, and Days 36 to 42
    E.5.2Secondary end point(s)
    1) Total number of bowel movements per week, after the first week of treatment
    2) Total number of bowel movements for the whole 2-week treatment period
    3) Mean daily stool form score according to the Bristol Stool Form Scale (BSFS), measured during the first week of treatment
    4) Mean daily stool form score according to the BSFS, measured during the second week of treatment
    5) Mean daily stool form score according to the BSFS, measured during the whole 2-week treatment period
    6) Total number of bowel movements with a BSFS score ≥ 6 during the first week of treatment
    7) Total number of bowel movements with a BSFS score ≥ 6 during the whole 2-week treatment period
    8) Mean daily dose of loperamide in mg, if used, during the second week of treatment
    9) Mean Diarrhea Composite Index ([weekly bowel movement frequency X mean daily BSFS score] + loperamide use [weekly mg X 3]) during the second week of treatment
    10) Safety (to be evaluated through the assessment of adverse events (AE), laboratory tests, vital signs, ECG and physical examination)
    11) Tolerability (to be evaluated through assessment of TEAEs and TESAEs)
    12) Evaluation of severity of diarrhea, abdominal pain, urgency and bloating using an 11-point Visual Analog Scale (VAS)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Days 1 to 7, and Days 29 to 35
    2) Days 1 to 14 and Days 29 to 42
    3) Days 1 to 7, and Days 29 to 35
    4) Days 8 to 14, and Days 36 to 42
    5) Days 1 to 14 and Days 29 to 42
    6) Days 1 to 7, and Days 29 to 35
    7) Days 1 to 14 and Days 29 to 42
    8) Days 8 to 14, and Days 36 to 42
    9) Days 8 to 14, and Days 36 to 42
    10) Throughout study
    11) Throughout study
    12) Days 0 (baseline), 15, 28 and 43
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Double-dummy
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Double-dummy crossover design - patients are their own controls and matching placebo is used
    E.8.2.4Number of treatment arms in the trial2
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned 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: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state18
    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 Decision2019-07-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-12-22
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