Based on our request for an update, Biogen has provided us with the following information on its global clinical study DEVOTE.
The study is designed to evaluate the safety, tolerability and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomised, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA.
The three-part study will include:
- An open-label safety evaluation cohort
- A pivotal, double-blind, active control randomised treatment cohort and
- An open-label treatment cohort.
After the safety evaluation cohort completes, the pivotal cohort will begin and include a treatment group receiving two loading doses of 50 milligrams (mg) 14 days apart, followed by a maintenance dose of 28 mg every four months.
A second treatment group will receive the current EU-approved administration of nusinersen, which is four loading doses of 12 mg followed by maintenance doses every four months. The third cohort will be an open-label evaluation to assess the safety and tolerability of transitioning patients from the currently approved dose of nusinersen to the higher dose being tested in the study.
The first patient has been treated in DEVOTE.