The DEVOTE trial is a phase II / III randomized, controlled, dose escalation trial that aims to assess the tolerance and efficacy of a higher dose of nusinersen (Spinraza®) in SMA than the one currently used. It will take place in nearly 50 countries, including in France. Biogen announced in a press release dated April 2, 2020 that the first participant in this trial, which will include 125 participants of all ages, has just received the injection of the product. Inclusion in France is expected to begin at the end of 2020.
Current dosage of nusinersen:
The recommended dose is 12 mg per administration.
Treatment with nusinersen consists of: 4 loading doses on days 0, 14, 28 and 63. A maintenance dose must then be administered every 4 months.
This trial takes place in 3 successive phases of 10 months:
- an evaluation phase of the safety of the open treatment: 28 mg of nusinersen for the first 3 loading doses then for the maintenance doses;
- a randomized, controlled, double-blind phase, comparing two groups: the high dose (50 mg on D1 and D15 then 28 mg on D135 and D279) versus the authorized dose of nusinersen (12 mg) according to the current protocol;
- an open treatment phase with the highest dose (50 mg on D1 then 28 mg on D121 and D241) for participants who have received the dose of 12 mg per injection for at least one year.
Biogen press release April 2,2020 « Biogen announces first patient treated with higher dose of Spinraza® (nusinersen) in phase 2/3 devote study »
On Clinicaltrials.gov : NCT04089566