Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, and potential for even greater efficacy of Spinraza (nusinersen) when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA). The Phase 2/3 randomized, 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 and a pivotal, double-blind, active control randomized treatment cohort followed by 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 U.S. Food and Drug Administration-approved administration of Spinraza, which is four loading doses with 12 mg 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 Spinraza to the higher dose being tested in the study.
More information on the study (NCT04089566) is available at clinicaltrials.gov.