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    Summary
    EudraCT Number:2021-002308-11
    Sponsor's Protocol Code Number:IM047-001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-10-22
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-002308-11
    A.3Full title of the trial
    A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
    A.4.1Sponsor's protocol code numberIM047-001
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1268-2191
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/233/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene International II Sàrl
    B.1.3.4CountrySwitzerland
    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 organisationBristol-Myers-Squibb International Corporation
    B.5.2Functional name of contact pointGSM-CT
    B.5.3 Address:
    B.5.3.1Street AddressParc de L'Alliance - Avenue de Finlande 4
    B.5.3.2Town/ cityBraine-l'Alleud
    B.5.3.3Post code1420
    B.5.3.4CountryBelgium
    B.5.6E-mailclinical.trials@bms.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 nameOzanimod HCL
    D.3.2Product code RPC1063
    D.3.4Pharmaceutical form 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 INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.23
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.46
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.92
    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
    Moderately to Severely Active Ulcerative Colitis
    E.1.1.1Medical condition in easily understood language
    Ulcerative Colitis
    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 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 evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical remission, Week 52)
    E.2.2Secondary objectives of the trial
    Please To evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical remission, Week 10)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Age ≥ 2 and < 17 years of age
    -Have had UC diagnosed prior to the Screening Visit. The diagnosis should be confirmed by clinical and endoscopic evidence and corroborated by a histopathology report
    -Evidence of UC extending beyond the rectum, as determined by baseline endoscopy
    -Has moderately to severely active UC, defined as a 4-component Mayo Score ≥ 6 and ≤ 12 at the time of screening, including the following: Mayo Endoscopy Subscore ≥ 2, Rectal Bleeding Score ≥ 1, and Stool Frequency Score ≥ 1.
    -Has had an inadequate response, loss of response to, or is intolerant to at least 1 of the following treatments for UC: oral aminosalicylates, systemic corticosteroids, immunomodulators, biologic therapy
    -Subjects may concurrently receive treatment with oral 5-ASAs, or with prednisone or equivalent (≤ 0.5 mg/kg/ day up to 20 mg/day).
    -Must have documentation of vaccinations per standard immunization schedule including complete Varicella zoster virus (VZV) vaccination at least 30 days prior to randomization, or documentation of positive Varicella zoster virus (VZV) Immunoglobulin G (IgG) antibody.
    -Females of childbearing potential (FCBP)[1] must agree to practice a highly effective method of contraception[2] throughout the study until completion of the 90-day Safety Follow-up Visit. Highly effective methods of contraception are those that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly. Acceptable methods of birth control in the study are the following:
    • combined hormonal (estrogen and progestogen containing) contraception, which may be oral, intravaginal, or transdermal
    • progestogen-only hormonal contraception associated with inhibition of ovulation, which may be oral, injectable, or implantable
    • placement of an intrauterine device (IUD)
    • placement of an intrauterine hormone-releasing system (IUS)
    • bilateral tubal occlusion
    • complete sexual abstinence (reliability to be evaluated based on the preferred and usual lifestyle of the subject)

    - Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception. Female condom and male condom should not be used together.
    E.4Principal exclusion criteria
    -Clinically relevant cardiovascular, hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the subject at risk by participating in the study.
    -Diagnosis of Crohn’s disease, indeterminate colitis or the presence or history of a fistula consistent with Crohn’s disease or microscopic colitis, radiation colitis or ischemic colitis.
    -Has documentation of positive test for toxin producing Clostridium difficile or PCR examination of the stool. If positive, subjects may be rescreened after appropriate treatment and retested no earlier than 7 days after completion of treatment.
    -Positive examination for pathogens
    -History of treatment with topical rectal 5-aminosalicylic acid or topical rectal steroids within 2 weeks of Screening endoscopy or anti-motility medications during screening.
    -Apheresis within 2 weeks of randomization.
    -Subjects with delayed growth or delayed pubertal development and who will not maintain a stable dose of corticosteroids through Week 5.
    -Pregnancy, lactation, or a positive serum beta human chorionic gonadotropin measured during Screening.
    -History or presence of the following clinically relevant cardiovascular conditions:
    Structural cardiac disease
    -Cardiac events or diseases that predispose to cardiac complications
    -Prolonged QT interval corrected for heart rate using Fridericia’s formula
    > 450 msec for both males and females (at either Screening or Day 1 Predose assessment), or is at additional risk for QT interval prolongation
    -During either Screening or Baseline Day 1 pre-dose assessment, resting HR < 55 bpm in subjects 12-17 years old, resting HR < 65 bpm in subjects 6-11 years old, or resting HR < 75 bpm in subjects 2-5 years old. Subjects will be allowed one recheck per visit
    -Severe untreated sleep apnea
    -History of diabetes mellitus type 1, or uncontrolled DM type 2 glycosylated hemoglobin or a diabetic subject with significant comorbid conditions.
    -History of uveitis within the last year prior to Screening, or history or evidence of retinal disease.
    -Subject has any known active bacterial, viral, fungal mycobacterial infection or any major episode infection that required hospitalization or treatment with IV antibiotics within 30 days of Screening or oral antibiotics within 14 days of screening.
    -In the case of prior SARS-CoV-2 infection, symptoms must have completely resolved and based on Investigator assessment in consultation with the Clinical Trial Physician, there are no sequelae that would place the subject at a higher risk of receiving investigational treatment.
    -Any history of cancer or colonic dysplasia that has not been completely removed.
    -History of or currently active primary or secondary immunodeficiency, or subjects with known genetic disorders as a cause for colitis
    -Serum creatinine > 1.4 mg/dL for females, or > 1.6 mg/dL for males

    -In subjects with recent pulmonary function test measurements: Forced expiratory volume at 1 second or a forced vital capacity at < 70% of predicted values
    -Exclusions related to medications
    -Hypersensitivity to active ingredients or excipients of ozanimod.
    -Treated with a biologic agent within 8 weeks or 5 elimination half-lives (whichever is shorter) prior to the first dose of IP
    -Undetectable drug levels on therapeutic drug monitoring assays may be utilized.
    -Treated with an investigational agent, including for COVID-19, within 5 elimination half-lives of that agent prior to the first dose of IP
    -Received a live or live attenuated vaccine within 4 weeks prior to the first dose of IP.
    -Received any previous treatment with lymphocyte-depleting therapies
    -Received treatment with D-penicillamine, leflunomide, or thalidomide within 8 weeks of first dose of IP
    -Received previous treatment with natalizumab, fingolimod, or other S1P receptor modulators
    -History of treatment with IVIg, or plasmapheresis within 3 months prior to first dose of IP.
    -Received previous treatment with oral cyclosporine, tacrolimus, sirolimus, or mycophenolate mofetil within 16 weeks of Screening.
    -Planned concurrent treatment with immunomodulatory agents after randomization.
    -Chronic nonsteroidal anti-inflammatory drug use
    -Receiving treatment with Class Ia or Class III anti-arrhythmic drugs or with 2 or more agents in combination known to prolong PR interval.
    -Receiving treatment with breast cancer resistance protein inhibitors
    -Received cholestyramine or other drugs interfering with enterohepatic circulation within 3 weeks of Screening.
    -Receiving treatment with any of the following drugs or interventions within the corresponding timeframe:
    At randomization:
    -CYP2C8 inhibitors and inducers
    Two weeks prior to randomization:
    -Monoamine oxidase inhibitors
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects who achieve clinical remission, defined as all the following:
    • Mayo Endoscopy Subscore (MES) ≤ 1
    • Rectal Bleeding Subscore (RBS) = 0
    • Stool Frequency Subscore (SFS) ≤ 1, with a decrease of ≥ 1 point from Baseline SFS
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    E.5.2Secondary end point(s)
    1/Proportion of subjects who achieve clinical response defined as all the following:
    • Decrease from Baseline in the 3-component Mayo score of at least 2 points and at least 35%
    • Decrease in RBS of at least 1 point OR absolute RBS ≤ 1
    2/Proportion of subjects who achieve clinical remission defined as all the following:
    • MES ≤ 1
    • RBS = 0
    • SFS ≤ 1, with a decrease of ≥1 point from Baseline SFS
    3/Proportion of subjects who achieve symptomatic improvement of UC, defined as all the following:
    • RBS = 0
    • SFS ≤ 1
    • Decrease in SFS of ≥ 1 from Baseline
    4/Proportion of subjects who achieve endoscopic improvement, defined as MES ≤ 1
    5/Proportion of subjects who achieve corticosteroid-free remission at Week 52, defined as all the following:
    • Did not receive steroids for ≥ 12 weeks prior to Week 52
    • Achieved clinical remission defined by the 3-component Mayo score
    6/Secondary – Safety and Tolerability
    Number and proportion of subjects experiencing AEs, SAEs, AEs leading to discontinuation from treatment, and AEs of interest (AEIs)
    7/Secondary - Pharmacokinetics
    Steady state systemic exposure of ozanimod and CC112273
    8/Secondary – Pharmacodynamics
    Absolute and percent change from baseline in ALC
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/Week 10 and week 52
    2/Week 10
    3/Week 10 and week 52
    4/Week 10 and week 52
    5/Week 52
    6/throughout the study
    7/Week 18 and throughout the study
    8/Week 10 and 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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    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) 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 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.8.2.4Number 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    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
    Israel
    Japan
    Russian Federation
    United States
    Belgium
    France
    Germany
    Netherlands
    Poland
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    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
    Last patient Last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years8
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days30
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 120
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 48
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 72
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 120
    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 who continue to demonstrate clinical benefit will be eligible to receive Celgene supplied study treatment for a maximum treatment duration as specified in Section 7.1. of the protocol.
    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 Decision2021-12-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-01
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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