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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

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

Detailed Summary:
Sponsor: Bioblast Pharma Ltd.

Current Primary Outcome:

  • vital signs [ Time Frame: 28 weeks ]
  • safety lab tests [ Time Frame: 28 weeks ]
  • Adverse Events [ Time Frame: 28 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome: Changes in disease markers [ Time Frame: 28 weeks ]

Original Secondary Outcome: Same as current

Information By: Bioblast Pharma Ltd.

Dates:
Date Received: December 8, 2013
Date Started: February 2014
Date Completion:
Last Updated: April 5, 2016
Last Verified: March 2015