Drug Combo Slows Functional Decline in Amyotrophic Lateral S
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Treatment with a fixed-dose combination of sodium phenylbutyrate and taurursodiol slows the rate of decline in physical function in patients with amyotrophic lateral sclerosis (ALS), according to results of the phase 2/3 CENTAUR study.

In this multicenter, randomized, double-blind trial, researchers enrolled participants with definite ALS who had had an onset of symptoms within the previous 18 months. Participants were randomly assigned in a 2:1 ratio to receive sodium phenylbutyrate–taurursodiol (3 g of sodium phenylbutyrate and 1 g of taurursodiol, administered once a day for 3 weeks and then twice a day) or placebo.

The primary outcome was the rate of decline in the total score on the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised through 24 weeks. Secondary outcomes were the rates of decline in isometric muscle strength, plasma phosphorylated axonal neurofilament H subunit levels, and the slow vital capacity; the time to death, tracheostomy, or permanent ventilation; and the time to death, tracheostomy, permanent ventilation, or hospitalization.

Results:
-- A total of 177 persons with ALS were screened for eligibility, and 137 were randomly assigned to receive sodium phenylbutyrate–taurursodiol (89 participants) or placebo (48 participants).
-- In a modified intention-to-treat analysis, the mean rate of change in the ALSFRS-R score was -1.24 points per month with the active drug and -1.66 points per month with placebo.
-- Secondary outcomes did not differ significantly between the two groups.
-- Adverse events with the active drug were mainly gastrointestinal.

Conclusively, Sodium phenylbutyrate–taurursodiol resulted in slower functional decline than placebo as measured by the ALSFRS-R score over a period of 24 weeks. Secondary outcomes were not significantly different between the two groups. Longer and larger trials are necessary to evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol in persons with ALS.

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1916945
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