Study Period | Screening | Adaptive PK study | Double-blind Treatment Period | Follow up | ||||
---|---|---|---|---|---|---|---|---|
Timing (weeks) | −4 to −1 | Day 1 | Day 4 | 2 (pre-dose) – 27 | 27–29 | |||
Window (days) | +/−7 (except +7 for V7) | +/− 2 | ||||||
Visit number | V1 | V2 | V3 | V4a | V5 | V6 | V7b | V8 |
Day/Week | −4 to −1 | Day 1 | Day 4 | Wk 2 / Day 1 | Wk 6 | Wk 13 | Wk 27 | Wk 29 |
Informed Consent | x | |||||||
Inclusion/Exclusion criteria | x | x | x | |||||
Demographics | x | xc | ||||||
Medical history | x | x | xc | |||||
Laboratoryd | x | x | xe | x | x | x | x | |
Pregnancy testf | x | x | x | x | Monthly | |||
Physical examination | x | x | x | x | x | x | ||
Bodyweight and heightg | x | x | xh | xh | x | |||
Vital signsi | x | x | xg | x | x | x | x | x |
ECGk | x | xj | xj | x | x | x | x | x |
2D-Echocardiographyl | x | x | ||||||
GMFM-88 | x | x | x | x | x | x | ||
IPMDS | x | x | x | x | x | x | ||
Study medication | Daily | |||||||
Randomisation | xm | |||||||
PEDI-CAT | xm | x | x | x | x | |||
BAD | xm | x | x | x | x | |||
Tardieu Spasticity test | xm | x | x | x | x | |||
SARA | xm | x | x | x | x | |||
9 Hole Peg Test | xn | xm | x | x | x | x | ||
10 MWT | xm | x | x | x | x | |||
Zarit-12 Burden scale | xm | x | x | x | x | |||
NeuroQL-SF | xm | x | x | x | x | |||
Clinician-scored and Patient/Caregiver scored GIC, MBSAo | x | x | x | x | x | |||
EQ-5D-Y | xm | x | x | x | x | |||
Health Utilities Index | xm | x | x | x | x | |||
Palatability | x | |||||||
Acceptability | Continuously | |||||||
PK samplingp | x | x | x | |||||
Telephone compliance Checksq | Weekly | |||||||
AE recording | Continuously | |||||||
Concomitant medication | Continuously | |||||||
Diaryr | Continuously | |||||||
Study medication dispensing | x | x | x | x | ||||
Return study medication and Drug accountability | x | x | x | x |