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Accepted for/Published in: JMIR Research Protocols

Date Submitted: Feb 23, 2023
Date Accepted: Nov 23, 2023

The final, peer-reviewed published version of this preprint can be found here:

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

Okamoto T, Ishizuka T, Shimizu R, Asahina Y, Nakamura H, Shimizu Y, Nishida Y, Yokota T, Lin Y, Sato W, Yamamura T

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

JMIR Res Protoc 2024;13:e46709

DOI: 10.2196/46709

PMID: 38224478

PMCID: 10825757

Warning: This is an author submission that is not peer-reviewed or edited.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

  • Tomoko Okamoto; 
  • Takami Ishizuka; 
  • Reiko Shimizu; 
  • Yasuko Asahina; 
  • Harumasa Nakamura; 
  • Yuko Shimizu; 
  • Yoichiro Nishida; 
  • Takanori Yokota; 
  • Youwei Lin; 
  • Wakiro Sato; 
  • Takashi Yamamura

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


 Citation

Please cite as:

Okamoto T, Ishizuka T, Shimizu R, Asahina Y, Nakamura H, Shimizu Y, Nishida Y, Yokota T, Lin Y, Sato W, Yamamura T

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

JMIR Res Protoc 2024;13:e46709

DOI: 10.2196/46709

PMID: 38224478

PMCID: 10825757

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