Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

NCT02015481 ↗

Phase 2 Completed 25 enrolled Bioblast Pharma Ltd.

NaSingle-groupOpen-labelTreatment

Summary

The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Timeline

Start: 2014-02
Primary completion: 2016-04
Completion: 2016-04

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Trehalose	Small molecule	30 g	Intravenous

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Summary

Timeline

Drugs

Indications