Accepted for/Published in: JMIR Research Protocols
Date Submitted: Feb 23, 2023
Date Accepted: Nov 23, 2023
Warning: This is an author submission that is not peer-reviewed or edited. Preprints - unless they show as "accepted" - should not be relied on to guide clinical practice or health-related behavior and should not be reported in news media as established information.
NKT Cell-stimulatory Glycolipid OCH-NCNP1 Protocol of a Randomized, Placebo-controlled Phase II Trial for Patients with Relapsing Multiple Sclerosis
ABSTRACT
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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