Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
Status:
Withdrawn
Trial end date:
2018-08-15
Target enrollment:
Participant gender:
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.