Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet

• Register: ClinicalTrials.gov
• Last refreshed on: 7 September 2015
• Main ID: NCT02383511
• Date of registration: 04/02/2015
• Prospective Registration: No
• Primary sponsor: Summit Therapeutics
• Public title: Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
• Scientific title: A Phase 1b Placebo-controlled, Multi-centre, Randomized, Double-blind Dose Escalation Study to Evaluate the Pharmacokinetics (PK) and Safety of SMT C1100 in Patients With Duchenne Muscular Dystrophy (DMD) Who Follow a Balanced Diet
• Date of first enrolment: February 2015
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02383511
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United Kingdom

Key inclusion & exclusion 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).



Age minimum: 5 Years
Age maximum: 13 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: SMT C1100

Primary Outcome(s)

Pharmacokinetic parameters at different dose levels of SMT C1100 [Time Frame: 28 days]

Secondary Outcome(s)

Pharmacokinetic parameters at different dose levels of SMT C1100 [Time Frame: 28 Days]

Evaluation of plasma CK levels [Time Frame: 42 days]

Safety and tolerability of SMT C1100 [Time Frame: 28 days]

Secondary ID(s)

SMT C11003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors