Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate in this study, where all subjects will be treated with a single dose of BB-301. BB-301 will be injected directly into the middle pharyngeal constrictor muscle and the inferior pharyngeal constrictor muscle of the throat through the use of an open surgical procedure conducted under general anesthesia. The primary objectives of the study are to evaluate the safety of BB-301, to identify the best dose of BB-301 to administer to patients, and to characterize how well BB-301 works to improve the symptoms of dysphagia in patients with OPMD.
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Incidence of dose-limiting toxicities (DLTs) in phase 1b
Timeframe: Up to 60 days
Incidence of adverse events (AEs) according to NCI CTCAE v5.0 in phase 1b and in phase 2a
Timeframe: Up to 360 days
Phase 1b: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Pharyngeal constrictor muscle function as estimated by the pharyngeal area at maximum constriction (PhAMPC)
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Pharyngeal constrictor muscle function as estimated by PhAMPC
Timeframe: Baseline, Day 90, Day 180, Day 270, Day 360