First Patient Enrolled in Phase 3 Study of Troriluzole for Spinocerebellar Ataxia

The first patient has been enrolled in a phase 3 study (NCT03701399) of troriluzole (Biohaven, New Haven, CT) to treat spinocerebellar ataxia (SCA). The primary outcome measure of the trial is improvement of symptoms as measured by a decreased score on the Modified Scale for the Assessment and Rating of Ataxia (SARA). The study plan is to include approximately 230 participants at approximately 22 clinical sites. Participants will be randomly assigned to receive 200 mg troriluzole or placebo daily for 48 weeks. 

To be eligible to enroll, patients must: 

• be willing to have genetic testing (provided by study if eligible) if they haven’t already;
• be medically stable with known or suspected SCA types 1, 2, 3, 6, 7, 8, or 10;
• have a Mini Mental State Examination score less than 24; 
• be able to walk 8 meters alone or with an ambulatory aid (not a support person); 
• have a screening score of 3 or more on SARA with
  • a score of 1 or more on gait subsection of SARA,
  • scores less than 4 for each individual item,
  • and screening and baseline scores that do not differ by 2 or more points;
• have no medical conditions that can otherwise explain their ataxia or interfere with SARA scoring;
• and have no active liver disease. 

Post-hoc data from an earlier phase 2b/3 study (NCT02960893) suggest patients with SCA types 1, 2, 3 or 6 treated with troriluzole for 48 weeks had slightly slower disease progression (-.34 points, 95% CI: -0.94-0.26) compared with untreated patients in a natural history cohort (+1.07 points, 95% CI: -2.22—0.60; P = .0007). In this analysis, treated and untreated patients were matched by age, sex, baseline SARA score, and SARA gait subscore. 

Gil L'Italien, PhD, Head of Global Health Economics and Outcomes Research at Biohaven stated, "The findings . . . are encouraging and provide further support for the potential long-term therapeutic benefit of troriluzole in patients with SCA. We are thrilled to have now enrolled the first patient in the Phase 3 study, which will more fully test the therapeutic potential of troriluzole in treating SCA over the course of 1 year."