Phase 3 Biotin Trial Underway for Primary, Secondary Progressive Multiple Sclerosis
Study will assess improvements in mobility and changes in disability in patients with MS.
MedDay Pharmaceuticals has launched a phase 3 trial to examine whether treatment with high-dose biotin (MD1003) eases disability and improves mobility in patients with non-relapsing primary or secondary progressive multiple sclerosis (MS).
Biotin is a form of vitamin B and plays an important role in energy production within cells, according to Multiple Sclerosis News Today. Investigators hypothesize that the compound has no anti-inflammatory action, and therefore, has no effect on relapses, but do believe that it treats disability by tackling neuronal loss.
The randomized, double-blind phase 3 SPI2 trial (NCT02936037) is expected to enroll 600 patients with MS, especially those with gait impairment. The study is currently recruiting participants from across the United States, Canada, and Europe.
Participants will be randomized to receive either a 100 mg of biotin or a placebo 3 times per day for 15 months. The investigators expect the results to be known by mid-2019.
“An interesting point with biotin is that it doesn’t seem to work in the short term,” Frédéric Sedel, CEO and cofounder of MedDay, told Multiple Sclerosis News Today. “When we start the drug, we start to see an effect after at least 9 months of treatment.”
The patients will be allowed to enter an open-label extension study following the randomization phase of the trial. They will receive 100 mg of biotin for an additional 12 months.
The primary objective of the study is to assess improvements in mobility and changes in disability, measured on the Expanded Disability Status Scale or a timed walking test of 25 feet. Additionally, the investigators will analyze other parameters, such as cognitive function, quality of life, and disease activity.
In 2016, results from an earlier phase 3 study of biotin was published in the Multiple Sclerosis Journal. The findings showed that biotin eased the progression of disability and improved mobility in 12.6% of patients compared with those who received a placebo. The treatment was well-tolerated and there were no serious adverse events reported.
“What we observed in patients was progressive improvement, which was very unusual, as you know­­—–patients with progressive MS, as with other neurodegenerative diseases, are not supposed to really improve,” Sedel told Multiple Sclerosis News Today.
Sedel noted that approximately 5000 patients with MS in France are taking biotin outside of the earlier and ongoing clinical trial under the temporary authorization of use or ATU.