Riluzole in Huntington's disease: a 3-year, randomized controlled study.
OBJECTIVE
We conducted a randomized double-blind trial of riluzole in Huntington's disease to investigate the efficacy of this antiexcitotoxic drug in slowing disease progression.


METHODS
The study included 537 adult patients with a clinical diagnosis of Huntington's disease confirmed by genotyping. Patients were randomized (2:1) to treatment with riluzole (50mg twice daily) or placebo for 3 years. Concomitant use of antichoreic medication was forbidden, and introduction of such medication was a predefined end point. The primary outcome measure was change in a combined score derived from the motor and total functional capacity subscores of the Unified Huntington's Disease Rating Scale. Safety was also evaluated.


RESULTS
A total of 379 patients completed the study (mean age, 47 [standard deviation, 9.5] years; 50% female patients). The principal reason for discontinuation was introduction of antichoreic medication. The median change from baseline in the combined score (primary outcome) for the "per protocol" population was 13.7 (95% confidence interval, 11.1-17.2) in the placebo group and 14.3 (95% confidence interval, 11.7-16.6) in the riluzole group. No intergroup difference in outcome could thus be demonstrated (p = 0.93, Mann-Whitney U test). No differences in secondary efficacy outcome variables were observed except for more frequent recourse to antichoreic medication in the placebo group. No unexpected adverse events were reported, and tolerability was acceptable.


CONCLUSIONS
No neuroprotective or beneficial symptomatic effects of riluzole in Huntington's disease were demonstrated.
Landwehrmeyer
G Bernhard
GB
Department of Neurology, University of Ulm, Ulm, Germany.
Dubois
Bruno
B
de Yébenes
Justo Garcia
JG
Kremer
Berry
B
Gaus
Wilhelm
W
Kraus
Peter H
PH
Przuntek
Horst
H
Dib
Michel
M
Doble
Adam
A
Fischer
Wilhelm
W
Ludolph
Albert C
AC
D000328:Adult; D000368:Aged; D001519:Behavior / Q000502:physiology; D003071:Cognition / Q000502:physiology; D003863:Depression / Q000209:etiology / Q000523:psychology; D005260:Female; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy* / Q000235:genetics / Q000523:psychology; D008297:Male; D008875:Middle Aged; D009460:Neurologic Examination; D018696:Neuroprotective Agents / Q000009:adverse effects / Q000627:therapeutic use*; D011569:Psychiatric Status Rating Scales; D019782:Riluzole / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D018696:Neuroprotective Agents; D019782:Riluzole