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    EudraCT Number:2014-003100-78
    Sponsor's Protocol Code Number:SMT_C11003
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-09-19
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2014-003100-78
    A.3Full title of the trial
    SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study
    to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet.
    A.4.1Sponsor's protocol code numberSMT_C11003
    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 SponsorSummit (Oxford) Limited
    B.1.3.4CountryUnited Kingdom
    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 supportSummit (Oxford) Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSummit (Oxford) Limited
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address85b Park Drive, Milton Park
    B.5.3.2Town/ cityAbingdon
    B.5.3.3Post codeOX14 4RY
    B.5.3.4CountryUnited Kingdom
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/591
    D.3 Description of the IMP
    D.3.2Product code SMT C1100
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNNot available
    D.3.9.1CAS number 945531-77-1
    D.3.9.2Current sponsor codeSMT C1100
    D.3.9.3Other descriptive nameSMT C1100
    D.3.9.4EV Substance CodeSUB44065
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 placeboOral suspension
    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
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    An inherited disease causing muscle degeneration.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the single and multiple oral dose PK of SMT C1100 and its metabolites in patients with DMD who follow a balanced diet
    E.2.2Secondary objectives of the trial
    (1) To determine the safety and tolerability of single and multiple oral doses of SMT C1100 and its metabolites in patients with DMD who follow a balanced diet.

    (2) To evaluate the diurnal variability in the steady state PK of SMT C1100.

    (3) To evaluate reductions in creatine phosphokinase as a potential pharmacodynamic (PD) marker of SMT C1100 activity and clinical benefit.

    Exploratory Objective
    To quantify putative biomarkers of disease activity and dietary metabolism from blood and urine
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients will be required to satisfy the following criteria at the screening visit (and at the beginning of each new Treatment Period) unless otherwise stated:

    1. Patients will be males of any ethnic origin with a genetic diagnosis of DMD.

    2. Children between 5 and 13 years of age.

    3. A parent/legal guardian must date and sign a written consent on behalf of the patient, according to International conference on Harmonisation (ICH) and local regulations. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.

    4. The patient is willing to give verbal or written age appropriate assent to participate.

    5. For safety reasons, the patient’s parent/legal guardian must have a good understanding of the English language, which the consent/assent forms are available, and understand the requirements for reporting of any AE to the Investigator.

    6. The patient has 6 months or more stable systemic (Patients using an intermittent regimen of steroid are allowed to be enrolled) corticosteroid therapy prior to Screening. Dose modifications for body weight are permitted.

    7. The patient or parent is willing to adhere to a balanced diet from 1 week prior to dosing until the end of the follow-up period.

    8. Patients must agree to not have sexual intercourse during the study treatment phases and until the end of their participation in the study.
    E.4Principal exclusion criteria
    Patients will be excluded from the study if they satisfy the following criteria at the screening visit unless otherwise stated:

    1. Enrolment or participation in any therapeutic clinical trial within the prior 3 months or 5 times the half-life (whichever is longer). Prior exposure to SMT C1100 is NOT an exclusion criterion.

    2. Known hypersensitivity to the excipients of the study drug (i.e. Poloxamer 188 [Lutrol F68], Methyl paraben, Propyl paraben, Hydroxypropylmethyl cellulose [Pharmacoat 645], Glycerol, Non crystallizing sorbitol [70%] [Neosorb 70/70B],
    Xanthan gum, Strawberry cream flavour [PHS-132963]) or a previous history of drug allergy.

    3. The patient or parent is unwilling to adhere to a balanced diet from 1 week prior to dosing until the end of the follow-up period.

    4. Is dairy or lactose intolerant, has an allergy to egg or nuts or any other dietary restrictions that might interfere with the conduct of the study.

    5. Is unable to refrain from eating cruciferous vegetables and barbecued (chargrilled) meat for the duration of the study.

    6. Use of prohibited medication within 5 half-lives prior to baseline assessments, unless otherwise stated in Section

    7. Need for mechanical ventilation.

    8. The patient experiences intermittent or continuous difficulties in swallowing.

    9. Non ambulatory.

    10. Any clinically significant acute illness within 4 weeks of the start of dose administration.

    11. Any comorbidity that, in the opinion of the Investigator, increases the risk of participating in the study.

    12. Symptomatic cardiomyopathy that in the opinion of the Investigator prohibits participation in this study.

    13. Abnormality in the 12-lead ECG at the Screening visit that, in the opinion of the Investigator, increases the risk of participating in the study.

    14. Any clinically significant medical condition, other than DMD that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., concomitant illness, severe reflux, psychiatric condition or behavioural disorder).

    15. The Patient smokes or has exposure to daily passive smoking (including parent/legal guardian, siblings) so as to minimise environmental factors causing CYP 1A induction.

    16. Excessive exercise (Investigator opinion).

    Following pre-dose assessments, patients may be excluded from the study for the following reasons:

    • Clinically significant vital signs or 12-lead ECG findings
    • Intercurrent illness or clinically significant adverse events since Screening
    • Deviation from study restrictions (see Section 6.2) will not be allowed except in prior agreement with Sponsor. Agreement may be given if in the opinion of the investigator and Sponsor these deviations will not interfere with the study procedures, compromise the safety of patients, or affect the study results. Any such deviations will be recorded in the deviation log, source data and documented in the trial master file (TMF) and clinical study report (CSR) at the end of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetic assessments
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1, day 14
    E.5.2Secondary end point(s)
    1. safety and tolerability assessments
    2. CPK levels
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Throughout the study
    2. Screening, days 1, 7, 14 and follow-up
    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 No
    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
    Exploratory biomarkers
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    Safety, tolerability and pharmacokinetics in patient population
    E.7.2Therapeutic exploratory (Phase II) No
    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 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 concerned4
    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
    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 months4
    E.8.9.1In the Member State concerned days0
    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: 12
    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. of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 2
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    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 Yes
    F. of subjects incapable of giving consent
    Subjects aged 5-13, parents/ legal guardians will be required to give
    Informed Consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    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)
    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 Decision2014-10-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-12-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-07-06
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