A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD) Patients Who Participated in Study BBCO-001

Trial Profile

A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD) Patients Who Participated in Study BBCO-001

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 24 Nov 2016

At a glance

  • Drugs Trehalose (Primary)
  • Indications Oculopharyngeal muscular dystrophy
  • Focus Therapeutic Use
  • Acronyms HOPEMD
  • Sponsors Bioblast Pharma
  • Most Recent Events

    • 21 Nov 2016 Planned End Date changed from 1 Dec 2016 to 1 Dec 2017.
    • 21 Nov 2016 Planned primary completion date changed from 1 Sep 2016 to 1 Aug 2017.
    • 26 Sep 2016 According to a Bioblast Pharma media release, results from this and a phase II study (Profile 700240232) are being presented at the annual scientific meeting of the Muscle Study Group Society (MSGS).
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