Efficacy and Safety of the Natural Killer T Cell–Stimulatory Glycolipid OCH-NCNP1 for Patients With Relapsing Multiple Sclerosis: Protocol for a Randomized Placebo-Controlled Clinical Trial

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Journal of Medical Internet Research - International Scientific Journal for Medical Research, Information and Communication on the Internet #Preprint #PeerReviewMe: Warning: This is a unreviewed preprint. Readers are warned that the document has not been peer-reviewed by expert/patient reviewers or an academic editor, may contain misleading claims, and is likely to undergo changes before final publication, if accepted, or may have been rejected/withdrawn. Readers with interest and expertise are encouraged to sign up as peer-reviewer, if the paper is within an open peer-review period. Please cite this preprint only for review purposes or for grant applications and CVs (if you are the author). Background: Multiple sclerosis (MS) is an autoimmune inflammatory disease of the central nervous system that causes myelin sheath damage and axonal degeneration. The glycolipid OCH-NCNP1 (OCH) exerts an immunoregulatory action that suppresses Th1 cell-mediated immune responses through natural killer T cell activation, selective interleukin-4 production, and Th2 bias induction in human CD4-positive natural killer T cells. Objective: This trial investigates the efficacy and safety of the immunomodulator OCH in patients with relapsing MS through 24-week repeated administration. The protocol was also designed to confirm exploratory biomarkers of T cells and natural killer cells that fluctuated in the Phase I trial, as well as efficacy-related clinical endpoints, including relapses, disability, magnetic resonance imaging changes, and their association with combined endpoints. Methods: This protocol describes a double-blind, multicenter, placebo-controlled, randomized, phase II clinical trial conducted in September 2019. The duration of the trial was 24 weeks. The participants were randomly assigned to either a placebo control group or an OCH-NCNP1 group for the 24-week duration. Outcome measures included the primary outcome of magnetic resonance imaging changes and secondary outcomes including annual relapse rate, relapse-free period, sustained reduction in disability occurrence rate, period until sustained reduction in disability, no evidence of disease activity, and exploratory biomarkers from phase I trials (such as gene expression, cell frequency, and intestinal and oral microbiome). Results: We plan to enroll 30 patients in the full analysis set. Enrollment was closed in June 2021, and the study analysis was planned to end in March 2023. Conclusions: This randomized controlled trial will determine whether OCH-NCNP1 is effective and safe in patients with MS as well as provide evidence for the potential of OCH-NCNP1 as a therapeutic agent for MS. Clinical Trial: ClinicalTrials.gov Trial registration ID#: NCT04211740

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