Phase II trial of arsenic trioxide for chronic GvHD meets its primary endpoint

On March 29, 2021, it was announced that the phase II GMED16-001 trial, evaluating an intravenous formulation of arsenic trioxide in patients with chronic graft-versus-host disease (cGvHD), met its primary endpoint.

This prospective, multicenter, non-randomized study evaluated the clinical efficacy of arsenic trioxide (a new selective immunosuppressive/anti-inflammatory drug for cGvHD) administered intravenously as first-line treatment in 21 patients with moderate to severe cGvHD. The primary endpoint of the study was to show an improvement in complete or partial remission rates at 6 months.

Patients received daily intravenous infusions of arsenic trioxide over a 4-week period, in combination with prednisone with or without cyclosporine. The use of arsenic trioxide led to a drastic reduction of the prednisone dose, starting at Week 6. Discontinuation of prednisone was planned once patients achieved a complete remission.

After the 6-month follow-up, the primary efficacy endpoint was met, with a 75% overall response rate. Sustained response was also observed at 12 months post-treatment in all patients.

Based on these promising results, arsenic trioxide is expected to advance into a phase III trial to confirm its efficacy.