A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT02056808
• Date of registration: 21/11/2013
• Prospective Registration: No
• Primary sponsor: Summit Corporation Plc.
• Public title: A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
• Scientific title: SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy
• Date of first enrolment: November 2013
• Target sample size: 12
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT02056808
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United Kingdom

Contacts

• Name: Stefan Spinty, MD
• Affiliation: Alder Hey Children's NHS Foundation Trust

• Name: Imelda Hughes, MD
• Affiliation: Central Manchester University Hospitals NHS Foundation Trust - Royal Manchester Childrens Hospital

• Name: Helen Roper, MD
• Affiliation: Heart of England NHS Foundation Trust - Heartlands Hospital

• Name: Franceso Muntoni, MD
• Affiliation: Great Ormond Street Hospital for Children NHS Foundation Trust

Key inclusion & exclusion criteria

Inclusion Criteria:

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

- Children between 5 and 11 years of age.

- 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.

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

- For safety reasons, the patient's parent/legal guardian must have a good
understanding of the English language, as this is the only language the
consent/assent forms are written in, and understand the requirements for reporting of
any adverse event to the Investigator.

Exclusion Criteria:

- Enrollment or participation in any therapeutic clinical trial within the prior 3
months or 5 times the half-life (whichever is longer).

- 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.

- Known hypersensitivity to the excipients of the study drug or a previous history of
drug allergy.

- Use of the following therapies is prohibited during the study and for at least 5
half-lives prior to the start of dose administration: Inducers of cytochrome P450
CYP1A2 (eg, carbamazepine, phenytoin, primidone, rifampin, omeprazole, and
barbiturates), and moderate and strong inhibitors of CYP1A2 (e.g., fluvoxamine,
ciprofloxacin, enoxacin, mexiletine; propafenone, zileuton). Substrates of CYP1A2
with narrow therapeutic windows (e.g., tacrine, theophylline, methadone, mexiletine).
Nicotine, including exposure to daily passive smoking to minimize cytochrome P450 CYP
1A induction. Chargrilled food, cruciferous vegetables, caffeine, tea, and any
xanthine containing foods, and drinks are prohibited from 36 hours prior to check-in
until final discharge from study. Herbal supplements and homeopathic preparations
(unless approved by medical monitor).

- Need for mechanical ventilation.

- Non ambulatory.

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

- Any co-morbidity that, in the opinion of the Investigator, increases the risk of
participating in the study.

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

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

- 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 behavioral disorder).

- Exposure to daily passive smoking (including parent/legal guardian, siblings) so as
to minimize environmental factors causing cytochrome P450 CYP 1A induction. For
information SMT C1100 is metabolized by cytochrome P450 CYP 1A.

- Excessive exercise (Investigator opinion).



Age minimum: 5 Years
Age maximum: 11 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: SMT C1100

Primary Outcome(s)

Safety and tolerability [Time Frame: After 10 days of treatment phase]

Secondary Outcome(s)

Pharmacokinetic parameters at different dose levels [Time Frame: After single oral dose and after 10 days of treatment phase]

Secondary ID(s)

SMT C11002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors