Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

Summary

BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.

Timeline

Start

2017-06-15

Primary completion

2018-02-15

Completion

2018-08-15

Drugs

Indications