Functional outcome measures as clinical trial endpoints in ALS.
The topiramate study was a 12-month randomized placebo-controlled trial in patients with ALS. Follow-up evaluation of the placebo group (n = 97) constituted a well-described cohort of patients with ALS, in whom multiple outcome measures were assessed at 3-month intervals. During the 12-month study period, the decline of forced vital capacity (FVC%) and ALS functional rating scale (ALSFRS) was linear, whereas the decline of maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores was curvilinear. Rates of FVC% and ALFRS decline, but not of MVIC-arm or MVIC-grip, were independent predictors of survival.
Traynor
B J
BJ
Neurology Clinical Trials Unit, Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. btraynor@partners.org
Zhang
H
H
Shefner
J M
JM
Schoenfeld
D
D
Cudkowicz
M E
ME
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000401:mortality / Q000503:physiopathology; D001132:Arm / Q000503:physiopathology*; D015415:Biomarkers; D005260:Female; D005632:Fructose / Q000031:analogs & derivatives* / Q000627:therapeutic use; D018737:Hand Strength; D006801:Humans; D007537:Isometric Contraction; D015993:Life Tables; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care / Q000379:methods*; D011237:Predictive Value of Tests; D016016:Proportional Hazards Models; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D012720:Severity of Illness Index; D016019:Survival Analysis; D000077236:Topiramate; D016896:Treatment Outcome; D014797:Vital Capacity
D015415:Biomarkers; D000077236:Topiramate; D005632:Fructose