The following is a summary of “High-dose pharmaceutical-grade biotin in patients with demyelinating neuropathies: a phase 2b open label, uncontrolled, pilot study,” published in the October 2023 issue of Neurology by Créange et al.
Researchers launched a retrospective study to assess the efficacy of high-dose pharmaceutical-grade biotin in patients with demyelinating neuropathies of diverse causes.
They conducted a Phase IIb open-label, uncontrolled, single-center pilot study with 15 patients (divided into three groups of five patients) who had chronic demyelinating peripheral neuropathy. This included chronic inflammatory demyelinating polyradiculoneuropathy, anti-myelin-associated glycoprotein neuropathy, and Charcot-Marie-Tooth 1a or 1b. High-dose pharmaceutical-grade biotin (100 mg taken orally thrice a day for up to 52 weeks) was utilized as the investigational product. The goal was a 10% relative improvement in two of four electrophysiological variables for the primary endpoint. Secondary endpoints assessed neuropathy, muscle function, sensation, gait, balance, and nerve excitability.
The results showed that a single patient attained the primary endpoint. Upon analyzing the entire group, several secondary endpoint parameters demonstrated improvement: Overall Neuropathy Limitations Scale (ONLS) score, Medical Research Council (MRC) sum score, Inflammatory Neuropathy Cause and Treatment (INCAT), 6-minute walk distance, strength-duration time constant, and rheobase. A notable correlation existed between the enhancement in the 6-minute walk distance and the strength-duration time. Concerning safety, there were 42 adverse events, three severe; however, none were attributed to the investigational product.
Investigators concluded that high-dose biotin improved sensory and motor function in patients with demyelinating neuropathies.
Source: bmcneurol.biomedcentral.com/articles/10.1186/s12883-023-03440-y
