Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: SMT C1100
Pharmaceutical Form: Oral suspension
INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
CAS Number: 945531-77-1
Current Sponsor code: SMT C1100
Concentra
Sponsors
Summit Corporation plc
Eligibility
Sex/Gender
Male
Inclusion criteria
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. Are the trial subjects under 18? yes Number of subjects for this age range: 12 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 6.2.1.2) within 5 half-lives prior to baseline assessments, unless otherwise stated in Section 6.2.1.2. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety and tolerability of single and multiple oral doses of SMT C1100 in patients with Duchenne Muscular Dystrophy (DMD).;Secondary Objective: 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.;Primary 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;Timepoint(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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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;Timepoint(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 | — |
Countries
United Kingdom
Contacts
Public ContactClinical Trial Information
Summit Corporation plc
Outcome results
None listed