Time and event schedule for clinical visits | |||||||
Visit | −1 | 0 | 1 | 2 | 3 | 4 | 5 |
Phase | Screening | Baseline | Treatment period | EOS | |||
Week | −4 | 0 | 6 | 12 | 24 | 36 | 40 |
Day | −28 to −1 | 1 | 42 | 84 | 168 | 252 | 280 |
ICF signed | X | ||||||
Eligibility review | X | X | |||||
Medical history and prior med assessment | X | ||||||
Physical examination | X | X | X | X | X | ||
Brief neurological examination | X | X | X | X | X | ||
Height assessment | X | ||||||
Weight assessment | X | X | X | X | X | ||
Urine drug test | X | ||||||
HIV/viral hepatitis screen | X | ||||||
Serum pregnancy test | X | ||||||
Urine pregnancy test | X | X | X | X | |||
Vital signs | X | X | X | X | X | ||
12-lead ECG | X | X | X | X | X | ||
Clinical laboratory (blood) | X | X | X | X | X | X | |
Urinalysis | X | X | X | X | X | ||
Concomitant medication | X | X | X | X | X | X | X |
Randomisation | X | ||||||
Dispense/return drug | X | X | X | X | |||
Schedule next visit | X | X | X | X | X | X | |
PD sampling (whole blood, plasma, urine) | X | X | X | X | |||
PK sampling (blood) | X | X | X | ||||
Adverse events | X | X | X | X | X | X | X |
Electromyography (eg, MUNIX, MUSIX, MScanFit) | X | X | X | X | |||
ALSFRS-R | X | X | X | X | X | ||
FVC | X | X | X | X | X | ||
ALSSQOL-SF | X | X | X | X | |||
Health utilisation form (eg, percutaneous endoscopic gastrostomy (PEG) tube, noninvasive ventiliation (NIV), wheelchair) | X | X | X | X | |||
Falls Questionnaire | X | X | X | ||||
PGI (patient global impression) | X | X | X | X | |||
CGI (clinical global impression) | X | X | X | X | |||
Columbia-Suicide Severity Rating Scale (C-SSRS) | X | X | X | X | X | X | X |
ALS, amyotrophic lateral sclerosis; ALSFRS-R, ALS Functional Rating Score-Revised; EOS, end of study; FVC, forced vital capacity; ICF, informed consent form; MUNIX, motor unit number index; MUSIX, motor unit size index; PD, pharmacodynamic; PK, pharmacokinetic.