Design of Phase 3 Studies Evaluating Vixotrigine for Treatment of Trigeminal Neuralgia

Mona Kotecha,1 William P Cheshire,2 Helen Finnigan,3 Kathryn Giblin,1 Himanshu Naik,1 Joanne Palmer,4 Simon Tate,4 Joanna M Zakrzewska5 1Biogen, Cambridge, MA, USA; 2Department of Neurology, Mayo Clinic Florida, Jacksonville, FL, USA; 3Biogen, Maidenhead, UK; 4Convergence Pharmaceuticals, a Biogen Company, Cambridge, UK; 5Facial Pain Unit and Pain Management Centre, University College London Hospitals NHS Foundation Trust/University College London, London, UKCorrespondence: Mona Kotecha Email Mona.Kotecha@biogen.comPurpose: Vixotrigine (BIIB074) is a voltage- and use-dependent sodium channel blocker. These studies will evaluate the efficacy and safety of vixotrigine in treating pain experienced by patients with trigeminal neuralgia (TN) using enriched enrollment randomized withdrawal trial designs.Patients and Methods: Two double-blind randomized withdrawal studies are planned to evaluate the efficacy and safety of vixotrigine compared with placebo in participants with TN (NCT03070132 and NCT03637387). Participant criteria include ≥ 18 years old who have classical, purely paroxysmal TN diagnosed ≥ 3 months prior to study entry, who experience ≥ 3 paroxysms of pain/day. The two studies will include a screening period, 7-day run-in period, a 4- or 6-week single-dose-blind dose-optimization period (Study 1) or 4-week open-label period (Study 2), and 14-week double-blind period. Participants will receive vixotrigine 150 mg orally three times daily in the dose-optimization and open-label periods. The primary endpoint of both studies is the proportion of participants classified as responders at Week 12 of the double-blind period. Secondary endpoints include safety measures, quality of life, and evaluation of vixotrigine population pharmacokinetics.Conclusion: There is a need for an effective, well-tolerated, noninvasive treatment for the neuropathic pain associated with TN. The proposed studies will evaluate the efficacy and safety of vixotrigine in t