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    EudraCT Number:2013-002115-99
    Sponsor's Protocol Code Number:SMTC11002
    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:2013-07-31
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2013-002115-99
    A.3Full title of the trial
    SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial to investigate if the drug SMT C1100 is safe and well tolerated in children with DMD when given orally one to three times a day.
    A.4.1Sponsor's protocol code numberSMTC11002
    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 Corporation plc
    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 Corporation plc
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSummit Corporation plc
    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.3.1Product nameSMT 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 INN5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
    D.3.9.1CAS number 945531-77-1
    D.3.9.2Current sponsor codeSMT 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
    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 14.1
    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 safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD).
    E.2.2Secondary objectives of the trial
    Secondary Objective

    To determine the single and multiple oral dose pharmacokinetics (PK) of SMT C1100 in patients with DMD.

    Exploratory Objective

    To quantify potential systemic activity biomarkers in blood and urine will be investigated to assess; 1) variability between individuals and 2) whether multiple doses of SMT C1100 has any impact on the variability.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients will be males of any ethnic origin with a genetic diagnosis of DMD.

    2. Children between 5 and 11 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, in which the consent/assent forms are available, and understand the requirements for reporting of any adverse event to the investigator.
    E.4Principal exclusion criteria
    1. Enrolment or participation in any therapeutic clinical trial within the prior 3 months or 5 times the half-life (whichever is longer).

    2. Initiation or change (other than dose modifications for body weight) of systemic corticosteroid therapy within 2 months prior to the start of dose administration or discontinuation of corticosteroids within 30 days prior to the start of dose administration.

    3. 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], Strawberry cream flavour [PHS-132963]) or a previous history of drug allergy.

    4. Use of prohibited therapy (Protocol Section within 5 half-lives prior to baseline assessments, unless otherwise stated in Section

    5. Need for mechanical ventilation.

    6. Non ambulatory.

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

    8. Any comorbidity that, in the opinion of the investigator, increases the risk of participating in the study.

    9. Symptomatic cardiac myopathy that in the opinion of the investigator prohibits participation in this study.

    10. Abnormality in the 12-lead ECG that, in the opinion of the investigator, increases the risk of participating in the study.

    11. 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, psychiatric condition or behavioural disorder).

    12. Exposure to daily passive smoking (including parent/legal guardian, siblings) so as to minimise environmental factors causing CYP 1A induction. For information SMT C1100 is metabolised by CYP 1A.

    13. Excessive exercise (Investigator opinion).

    Following pre-dose assessments on Day -1 and Day 1, 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
    • Deviation from study restrictions (see Protocol 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 TMF and CSR at the end of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and Tolerability Assessments

    1) Adverse Event Reporting

    2) Vital Signs (Blood pressure and pulse rate and oral/ tympanic body temperature)

    3) 12 Lead ECG

    4) Clinical Laboratory Evaluations

    5) Physical Examination

    6) Body weight
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Day -1 then on-going

    2) Screening; Days 1 and 11: Pre-dose, 2, 4 and 8 hours post dose; Days 2, 7 and 12: morning

    3) Screening; Day -1; Days 1 and 11: Pre-dose and 4 hours post-am dose; Day 7: morning

    4) Screening; Day -1; Days 7 and 12: morning

    5) Screening; Days 2 and 12 morning

    6) Day -1; Day 12 morning
    E.5.2Secondary end point(s)
    Biomarker Assessments

    1) Blood muscle regerenration biomarkers

    2) Urine muscle regerenration biomarkers

    Pharmacokinetic Assessments

    3) Blood sampling for SMT C1100

    4) Metabolite analysis- Urine
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Days 1 and 11: pre-dose

    2) Day 11: 0 to 8 h

    3) Day 1: 0, 1, 2, 3, 4, 6, 9, 12 and 24 h post dose;
    Day 11: 0, 1, 2, 3, 4, 6, 9, 12 and 24 h post dose

    4) Day 11: 0-8hrs
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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) 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 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.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days16
    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: 12
    F.1.1.6Adolescents (12-17 years) No
    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 5-11, 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 Decision2013-08-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-04-23
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