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Ataxia Clinical Trial Progresses to 48-Week Extension Phase


As NAF previously reported, BioHaven Pharmaceutical Holding Company Ltd. recently completed its 8-week trial of dosing for its drug compound trigriluzole. This is the largest clinical trial for Spinocerebellar Ataxia (SCA) to-date. During the 8-week randomization phase, either the compound or a placebo was given to 141 adults across the U.S. with SCA. Recipients were unaware which they were receiving during this portion of the trial. This phase of the trial compared trigriluzole to the placebo, measuring their impact on Ataxia symptoms.

On October 2, 2017, BioHaven released topline data indicating that trigriluzole did not differentiate from the placebo in treating Ataxia symptoms, according to SARA scores, during the initial 8-week phase of the trial. A higher than expected rate of placebo response may have hampered the ability of the study to detect a signal and Biohaven is performing additional analyses to better understand the effects of trigriluzole on Ataxia.

The trial did show promising results for safety and tolerability of the compound. BioHaven remains hopeful that trigriluzole will have positive outcomes as a long-term nerve-protecting agent, thereby slowing the progression of Ataxia. BioHaven reaffirmed their commitment to the Ataxia community, as well as their compound, with their plans to see the study through to completion of the 48-week extension phase.


In the 48-week extension phase, all participants will take trigriluzole. The Ataxia progression of participants will be monitored regularly and compared to what would be expected based upon the Ataxia Natural History Study. These results will be analyzed to determine if the medication slows the rate of progression of the disease.

  • Trigriluzole, thus far, appears to be safe to take and well tolerated.
  • This trial had unexpectedly high reports of improvement in Ataxia symptoms for persons taking the placebo, making it difficult to accurately determine whether the medication is effective or not.
  • The topline data results do NOT affect the potential for success in the next phase of the trial.
  • A similar clinical trial, for a similar compound, was found to slow the progression for ALS (also called Lou Gehrig’s Disease) but did NOT have any effects on symptoms.
  • The pharmaceutical industry, as a whole, is better equipped to prepare for Ataxia clinical trials in the future because of this trial.
  • This trial proved to the pharmaceutical industry that the Ataxia clinical sites are prepared and competent for clinical trials, potentially encouraging them to investigate applications for Ataxia for their compounds.

Read BioHaven’s Press Release (links to PDF)

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