A phase I study of TAS-205 in patients with Duchenne muscular dystrophy.

ABSTRACT:

Objective

Currently, the only approved standard Duchenne muscular dystrophy (DMD) treatment in Japan is oral steroids, which have various disadvantages. Previous work has suggested that hematopoietic-type prostaglandin D synthase (HPGDS), involved in production of the inflammatory mediator prostaglandin D₂ (PGD₂), might have a role in DMD pathology. We therefore investigated the safety, pharmacokinetics (PK), and pharmacodynamics of a highly selective HPGDS inhibitor (TAS-205) in Japanese patients with genetically confirmed DMD.

Methods

This was a double-blind, randomized, placebo-controlled phase I study to evaluate the use of single or 7-day repeated doses of TAS-205 administered orally. The urinary excretion of PGD₂ metabolites was also as

SUBMITTER: Takeshita E 

PROVIDER: S-EPMC6243382 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature