Randomized trial demonstrates improved ataxia rating and functional scores with DL-3-n-butylphthalide in spinocerebellar ataxia type 3, indicating potential clinical benefit and safety.
Key Points
To evaluate the clinical efficacy and safety of oral DL-3-n-butylphthalide (NBP) over 12 months in patients with spinocerebellar ataxia type 3 (SCA3).
Randomized patients (1:1) to oral NBP (200 mg three times daily) or matching placebo for 12 months.
Evaluated primary outcomes in the modified intention-to-treat (mITT) population (N=116; NBP n=56, placebo n=60) using a mixed-effect model to measure 12-month changes in the Scale for Assessment and Rating of Ataxia (SARA) and the Spinocerebellar Ataxia Functional Index (SCAFI).
NBP significantly reduced ataxia progression on the SARA compared to placebo (estimated marginal mean difference: -0.86, 95% CI: -1.56 to -0.16, P = 0.02).
Functional capacity measured by SCAFI significantly improved in the NBP group relative to placebo (estimated marginal mean difference: 0.16, 95% CI: 0.01 to 0.31, P = 0.03).
Overall adverse event rates were 23.2% (13/56) with NBP versus 15.0% (9/60) with placebo (P = 0.26), with drug-related adverse events occurring in 21.4% (12/56) versus 10.0% (6/60; P = 0.09) and no serious adverse events reported in either group.