ALS: edaravone found safe, effective in well-defined group

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Takeaway

  • Edaravone (Radicava) slowed the decline in function during 24 wk in patients with early-stage definite or probable amyotrophic lateral sclerosis (ALS).

Why this matters

  • ALS is incurable and has limited treatment options.

Key results

  • Patients in the edaravone group had a smaller reduction in Revised ALS Functional Rating Scale (ALSFRS-R) score than counterparts in the placebo group (−5.01 vs −7.50; least-squares mean difference, 2.49; P=.0013).
  • The rate of treatment-emergent adverse events was high and identical in the 2 groups, at 84%.
  • Patients in the edaravone group had a lower rate of serious adverse events (16% vs 24%) and events leading to withdrawal (1% vs 6%).

Study design

  • A phase 3, randomized, double-blind trial among 137 patients aged 20-75 y with definite or probable ALS and disease duration of 2 y or less, from 31 hospitals in Japan.
  • Patients were randomly assigned to intravenous edaravone or placebo for 6 cycles and a total treatment duration of 24 wk.
  • Main outcome was change in ALSFRS-R score.
  • Funding: Mitsubishi Tanabe Pharma Corporation.

Limitations

  • Effect on long-term survival was not assessed.
  • The trial excluded patients with moderate to severe renal impairment.