Investigator–initiated phase 2 study of OCH–NCNP1 in patients with recurrent multiple sclerosis

Abstract

Diminished regulatory component of the immune system are reported in multiple sclerosis (MS). Synthetic glycolipid OCH acts on iNKT cells to selectively produce IL–4, alleviating experimental autoimmune encephalomyelitis an MS animal model. The first–in–human Phase I investigator–initiated clinical trial was conducted at our hospital during 2012–2017. We have confirmed tolerability of OCH in both HS and MS patients. Pharmacokinetics study revealed that absorption of OCH from intestine was better in human than primates and rodents. Moreover, we have observed several immune–related changes in the blood of the HS and MS. Phase II clinical trials were started in November 2019. We herein outline a single–center, two–group, randomized, double–blind, phase II clinical trial among 30 patients with recurrent multiple sclerosis (RMS) who received weekly, repeated oral administration (24 weeks). Test drug (0.3–g granules ; containing 3.0mg OCH–NCNP1) or a control drug (placebo) is administered to examine drug efficacy and safety. As of January 2021, 25 patients participated in the trial.