Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Code: SMT C1100
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Pending
CAS Number: 945531-77-1
Current Sponsor code:
Sponsors
Summit (Oxford) Limited
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Patients will be required to satisfy the following criteria at the screening visit: 1. Be able to provide written informed consent/assent as per local requirements. 2. Be male. 3.Be aged =5 years =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients will be excluded from the study if they satisfy the following criteria at the screening visit 1. Have physical exam findings that in the investigator’s opinion should be exclusionary e.g., lower limb injury that may affect 6MWD performance. 2. Have any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to the start of study treatment. 3. Have uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D). 4.Have abnormal GLDH at baseline (>1.5 x ULN) 5.Have abnormal coagulation times at baseline (>1.5 x ULN) 6. Have an abnormal ECG e.g., a QTcF >500ms, left bundle-branch block or any other major conduction defect. 7. Use beta blockers (however, if during the course of the study they are clinically indicated they can be initiated; not applicable for cohort 3 ) 8. Use herbal supplements and be unwilling to stop these for the duration of the study. 9. Have a known hypersensitivity to any of the ingredients or excipients of the IMP. (microfluidised oral suspension F3, cohort 1 : Poloxamer 188, Methylparaben, Propylparaben, Hydroxypropylmethyl cellulose, Glycerol, Non crystallizing sorbitol [70%], Xanthan gum, Strawberry cream flavour [PHS-132963]); 10.Have a known hypersensitivity to any of the ingredients or excipients of the IMP. Powder for oral suspension (F6): hypromellose acetate (not applicable to cohorts 1 and 3 ) 11. Have been exposed to another investigational drug or DMD interventional agent within 3 months prior to start of the Treatment Phase. Prior exposure to SMT C1100 or participation in an approved deflazacort access program (e.g FOR-DMD or ACCESS DMD clinical trials ) within this period would not exclude the patient (provided they have been on stable treatment for 6 months ) 12. Have a history of major surgical procedure within 12 weeks prior to the start of the Treatment Phase (week 1) 13. Be undertaking ongoing immunosuppressive therapy (other than corticosteroids). 14. Have an expectation of a major surgical procedure (e.g., scoliosis surgery) during the 12-month Treatment Phase of the study. 15. Require daytime ventilator assistance. 16. Have a prior or ongoing medical condition (e.g., concomitant illness, psychiatric condition, behavioural disorder, alcoholism, drug abuse), medical history, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results. 17. Be dairy or lactose intolerant or have any other dietary restrictions that might interfere with the conduct of the study. 18. Be a smoker, use other tobacco or nicotine products or be exposed to daily passive smoking (including parent/legal guardian, siblings) so as to minimize environmental factors causing CYP1A induction. 19.Be using an approved DMD medication or anticipates using one during the duration of t
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: From baseline to Weeks 12, 24, 36 and 48 ; Main Objective: To investigate changes in leg MRI in paediatric patients with DMD, following treatment with SMT C1100(cohorts 1 and 2 ) To investigate the relationships between changes in leg MRI with plasma concentrations of SMT C1100 and its metabolites in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2) To assess the safety and tolerability of SMT C1100 and its metabolites in paediatric patients with DMD ; Secondary Objective: To investigate changes in utrophin expression and muscle fibre regeneration in muscle, in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2 ) To investigate the relationships between changes , utrophin expression and fibre regeneration in muscle and safety parameters with plasma concentrations of SMT C1100 and its metabolites in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2 ) To investigate changes in pulmonary function tests in paediatric subjects with DMD, following treatment with SMT C1100 To investigate the relationships between changes in pulmonary function tests with plasma concentrations of SMT C1100 and its metabolites in paediatric subjects with DMD, following treatment with SMT C1100 ; Primary end point(s): Change from baseline to Weeks 12, 24, 36 and 48 in MRI leg muscles parameters(Cohorts 1 and 2) SMT C1100 and metabolite plasma concentrations at Weeks 1 (Days 1 and 7) as applicable (Cohorts 2 and 3 only) 4, 8 (Cohorts 1 and 2 only), 12, 24, 36 and 48 • Safety data including: Treatment emergent AEs | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from baseline to Week 24 or 48 in utrophin expression via muscle biopsy analysis (Cohorts 1 and 2) • Change from baseline to Week 24 or 48 in muscle regeneration biomarkers, via muscle biopsy analysis(Cohorts 1 and 2) • Change from baseline to Weeks 12, 24, 36 and 48 (Cohorts 1 and 2) and from baseline to Weeks 1, 24 and 48 (Cohort 3) in pulmonary function tests • Safety data including: • Vital signs (systolic and diastolic blood pressure and heart rate) • Physical examination • Twelve-lead electrocardiogram (ECG) • Echocardiogram (ECHO) • Pulmonary function tests (forced expiratory volume in 1 second [FEV1], forced vital capacity [FVC] maximum inspiratory pressure [MIP], maximum expiratory pressure [MEP] and peak expiratory flow [PEF]);Peak cough flow (PCF) (Cohort 3)Sniff nasal inspiratory pressure (SNIP) (Cohort 3 • Safety laboratory evaluations (clinical chemistry, haematology (all 3 cohorts) coagulation (Cohort 2 and Cohort 3) parameters and urinalysis (all 3 cohorts) ;Timepoint(s) of evaluation of this end point: From baseline to Week 24 or 48 | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Summit (Oxford) Limited
Outcome results
None listed