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
- 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).