Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia

The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).

The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.

Key Inclusion Criteria:

• Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
• Ability to ambulate 8 meters without assistance (canes and other devices allowed)
• Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
• Score of ≥ 2 on the gait subsection of the SARA
• Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed

Key Exclusion Criteria:

• Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the participants symptoms of ataxia
• Mini Mental State Exam (MMSE) score < 24
• SARA total score of > 30 points at screening
• Clinical history of stroke
• Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant