A Phase 3 clinical trial has been launched by MedDay Pharmaceuticals to investigate whether treatment with high-dose biotin (MD1003) may ease disability and improve mobility in non-relapsing primary or secondary progressive MS patients. The study is recruiting participants across the U.S., Canada and Europe.
Biotin is a form of vitamin B, and it plays an important role in energy production within cells. Researchers believe the compound has no anti-inflammatory action, and thus no effect on relapses, but that it treats disability by tackling neuronal loss.
Eligible patients must be 18 to 65 years old, and can maintain existing disease-modifying therapies, if treatment has been stable for at least three months before enrollment. A list of U.S. cities with testing sites can be viewed here.
This randomized and double-blinded trial, called SPI2 (NCT02936037), is expected to enroll 600 MS patients, especially those with gait impairment, who will be assigned to receive either a capsule of MD1003 (100 mg) or a placebo three times a day for 15 months. Results are expected to be known by mid-2019.
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“An interesting point with biotin is that it doesn’t seem to work in the short term,” Frédéric Sedel, MedDay’s CEO and co-founder, said in a recent interview with Multiple Sclerosis Today. “When we start the drug, we start to see an effect after at least nine months of treatment.”
Following the trial’s randomization phase, patients will be allowed to enter an open-label extension study in which all will receive treatment with MD1003 (100 mg) for an additional 12 months.
The study’s main objective is to assess improvements in mobility and changes in disability, as measured on the Expanded Disability Status Scale (EDSS) or a timed test of walking 25 feet. Researchers will also analyze other clinical parameters, such as cognitive function, quality of life, and disease activity as captured on magnetic resonance imaging (MRI) scans.
Results of an earlier Phase 3 clinical trial (NCT02220933) of MD1003 in 154 patients with primary or secondary progressive MS in France, published in the Multiple Sclerosis Journal in 2016, found eased disability progression and improved mobility in 12.6 percent of treated patients, compared to those taking a placebo. The treatment was well-tolerated and no serious side effects were 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 said in the interview.
About 5,000 MS patients in France are also taking MD1003 outside of the earlier and ongoing clinical study, under a temporary license, known as an ATU, granted the treatment by French regulatory authorities, Sedel said.
More information about the newly starting Phase 3 trial is available on its clinical trials.gov webpage.