HOPEMD Phase 2 Open-Label Study
Evaluating a promising new treatment for patients with OPMD
The primary objective of the HOPEMD Phase 2 six-month open-label clinical study was to assess the safety and tolerability of trehalose 90 mg/mL IV solution. Although not powered for efficacy, secondary endpoints were included to explore if trehalose 90 mg/mL IV solution could improve or prevent worsening of OPMD disease markers.
The study enrolled 25 patients with clinical dysphagia and muscle weakness at two centers in Canada and Israel. All patients have completed 24 weeks of weekly treatment with trehalose 90 mg/mL IV solution.
Trehalose 90 mg/mL IV solution was observed to be safe and well-tolerated with no drug-related serious adverse events.
|Elevated urine glucose*||52||13|
|Urinary tract infection||12||3|
|Pain in extremity||12||3|
No patients withdrew due to adverse event.
Most adverse events were considered mild to moderate.
|Urinary tract infection||1||Resolved|
Three adverse events occurred in 2 patients.
Improvements versus baseline were observed in multiple secondary efficacy endpoints related to dysphagia and muscle strength and function.
Based on the positive safety and efficacy signals, Bioblast Pharma intends to enroll more than 70 patients in a Phase 2b double-blind placebo-controlled clinical trial, at up to 15 sites in the United States and Canada, to confirm the results from HOPEMD.
Find additional information about the results of the HOPEMD Phase 2 study.
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