Treatment emergent adverse events: all causalities.

<p>Treatment emergent adverse events: all causalities.</p

Baseline patient characteristics.

<p>Baseline patient characteristics.</p

Plot of the average reduction from baseline (estimate, square) of the following plasma markers: creatine phosphokinase (CPK), alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALK), gamma-glutamyl transferase (GGT), and albumen (ALB).

<p>Upper and lower 95% confidence intervals for the following time points after dosing shown: Day 7, Day 12, and follow-up (3 days after completion of dosing).</p

Individual and mean pharmacokinetics for SMT C1100 following multiple (Day 11) oral doses.

<p>Individual and mean pharmacokinetics for SMT C1100 following multiple (Day 11) oral doses.</p

Individual patient waterfall plots of gamma-glutamyl transferase (GGT), aspartate aminotransferase (AST), creatine phosphokinase (CPK), and alanine aminotransferase (ALT) showing changes from baseline after 10 days of dosing with SMT C1100.

<p>Individual patient waterfall plots of gamma-glutamyl transferase (GGT), aspartate aminotransferase (AST), creatine phosphokinase (CPK), and alanine aminotransferase (ALT) showing changes from baseline after 10 days of dosing with SMT C1100.</p

Individual and mean pharmacokinetics for SMT C1100 following single (Day 1) oral doses.

<p>Individual and mean pharmacokinetics for SMT C1100 following single (Day 1) oral doses.</p

Comparison of the average plasma levels of SMT C1100 from the 10 Duchenne muscular dystrophy pediatric patients (DMD fed) who had unexpectedly low exposure to SMT C1100 compared with the average plasma level of five healthy volunteers (HV) fasted prior to a single 200 mg/kg dose of SMT C1100.

<p>Comparison of the average plasma levels of SMT C1100 from the 10 Duchenne muscular dystrophy pediatric patients (DMD fed) who had unexpectedly low exposure to SMT C1100 compared with the average plasma level of five healthy volunteers (HV) fasted prior to a single 200 mg/kg dose of SMT C1100.</p

Study flow chart showing a multiple ascending dose design.

<p>Study flow chart showing a multiple ascending dose design.</p

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy - Fig 2

<p>Individual plasma concentrations of SMT C1100 for each of four patients in Groups A (black line), B (red line) and C (blue line). Group A patients received a single 50 mg/kg dose on Day 1 and on the morning of Day 11, with 50 mg/kg bid on Days 2 to 10. Group B and Group C patients received a single 100 mg/kg dose on Day 1 and on the morning of Day 11, with 100 mg/kg bid (Group B) or 100 mg/kg tid (Group C) on Days 2 to 10.</p

Additional file 1: of A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial

Checklist supplementary information. Supplementary information to implementing an international, multi-centre, academic-led clinical trial: checklist. (DOCX 115 kb