Oculopharyngeal Muscular Dystrophy Clinical Trial
Official title:
A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of
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.
After signing informed consent, patients will undergo two rounds of ice-cold water and nectar
drinking tests at least 1 week apart to confirm oropharyngeal dysfunction. Patients who have
confirmed oropharyngeal dysfunction, i.e., an ice-cold water drinking test time of 8 seconds
or greater at both rounds, in addition to an SSQ score of >235, will be enrolled. Baseline
values for all safety and efficacy parameters will be established during the screening
period. Patients will be randomized in a 1:1 ratio, to trehalose or placebo, at the time of
enrollment. Randomization will be stratified according to the patient's score on the SSQ at
screening (≤ 799 or ≥ 800).
Patients randomized to trehalose will receive a 1-hour IV infusion of trehalose at a dose of
0.75 g/kg weekly for 24 weeks. Patients randomized to placebo (normal saline) will receive a
weight-based equal volume of placebo weekly for 24 weeks.
After Week 24, patients may transition to an open-label extension of the study (extension
period). During the extension period, patients will be treated with weekly infusion of
trehalose at a dose of 0.75 g/kg for 24 weeks, followed by a 4-week safety follow-up (total
duration of study = 56 weeks).
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