Clinical trial NCT02383511 (phase 1, status: completed)
Conditions: muscular dystrophy, duchenne
ICD codes: G71.01
Drugs: smt c1100, smt c1100, smt c1100
SMILES: CCS(=O)(=O)C1=CC=C2OC(=NC2=C1)C1=CC=C2C=CC=CC2=C1, CCS(=O)(=O)C1=CC=C2OC(=NC2=C1)C1=CC=C2C=CC=CC2=C1, CCS(=O)(=O)C1=CC=C2OC(=NC2=C1)C1=CC=C2C=CC=CC2=C1
Eligibility criteria:

        Inclusion Criteria:

          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.

        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). Prior exposure to SMT C1100 is NOT an
             exclusion criterion.

          2. Known hypersensitivity to the excipients of the study drug 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 protocol.

          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).