Safety and pharmacokinetics of single and repeated dose of a novel formulation of intra-nasal Oxytocin (TTA-121) in healthy Japanese volunteers (double blind, placebo-controlled Phase 1 trial)

Abstract

Background:

Autism spectrum disorder (ASD) is a neurodevelopmental disorder, mainly exhibit problems in social communication/interaction behaviors. ASD is shown to affect one out of 100 individuals, however, the cause and the established treatments on the core symptoms of ASD are unavailable. Recently, peptide hormone Oxytocin showed some promises in treating ASD core symptoms, and various clinical studies are currently underway. The objective of this study is to evaluate the safety and pharmacokinetics of new formulation of intra-nasal Oxytocin in healthy Japanese male.

Methods:

In this double-blind, randomized, placebo-controlled Phase1 study, 5 cohorts of eight subjects received a single dose of intranasal TTA-121 at dose levels of 5, 10, 30, 100 and 200 U/ml or placebo and 3 cohorts of eight subjects received 30, 100 and 200 U/ml or placebo at repeated doses for 9 days. Safety assessments were conducted throughout the study.

Results:

Regarding safety, no serious adverse events were reported and no clinically significant findings were observed throughout the study. A linear relationship between plasma concentration of Oxytocin and administered dose of TTA-121 (5 to 200 U/ml) was observed. There was no drug accumulation after multiple doses of intra-nasal Oxytocin.

Conclusions:

TTA-121 was well tolerated and safe in Japanese healthy male subjects after administration of multiple doses up to 200U/ml, BID for 9 days.

Trial Registration:

UMIN000025922