An exploratory Phase 1/2 clinical trial in primary progressive multiple sclerosis (PPMS) conducted at the National Institutes of Health (NIH) confirms the safety profile of Raxone (idebenone) at a dose of 2,250 mg daily over two years.
But no difference in effectiveness was found between the Raxone-treated group and the placebo group.
The announcement was made by Santhera Pharmaceuticals, the company developing Raxone.
The trial (NCT00950248) is a double-blind, placebo-controlled study assessing the safety and efficacy of Raxone in PPMS. It is sponsored by the National Institute of Neurological Disorders and Stroke (NINDS), part of NIH.
The initial aim was to assess the safety, effectiveness, and mechanism of action of Raxone in 85 patients with PPMS over two years. A total of 77 patients were eventually randomized, and 66 completed the trial (33 in the active group and 33 in the placebo group).
The entire trial period combined a one-year observational pre-treatment phase and a two-year treatment period.
There was no difference between Raxone and placebo groups in the occurrence and severity of adverse events.
The study’s primary outcome was to explore the efficacy of Raxone through change in the CombiWISE scale — a disability rating system developed by NINDS investigators.
We are sorry that this post was not useful for you!
Let us improve this post!
Tell us how we can improve this post?