Muscular Dystrophy, Oculopharyngeal (OPMD) Clinical Trial
Official title:
A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent control, parallel group study. Patients completing protocol BBCO-001 will be offered the opportunity to enter into this 12-month randomized withdrawal protocol.
This will be a multi-center, randomized withdrawal, open-label, non-treatment concurrent
control, parallel group study. Patients completing protocol BBCO-001 will be offered the
opportunity to enter into this 12-month randomized withdrawal protocol.
Eligible patients will be randomized to one of the following treatment arms:
- Treatment Arm 1: active treatment; continuation of 30 g IV Cabaletta once a week over an
additional 52 weeks.
- Treatment Arm 2: no-treatment concurrent control; discontinuation of IV Cabaletta and
follow-up over 52 weeks.
IV Cabaletta will be administered once a week to patients in Treatment Arm 1. All patients,
regardless of treatment arm allocation, will undergo the same safety and efficacy assessments
during the monthly site visits.
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