A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
← catalyst calendarNCT06185673 · readout ≤ 1,564 d
Sponsored by Benitec Biopharma, Inc. (industry) · BNTC — their whole pipeline →.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
30estimated
Sites
1
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2023-11-28 | actual | When the study began enrolling |
| Primary completion | Nov 2030 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Nov 2040 | estimated | The whole study's end, after follow-up |
| First posted | 2023-12-29 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-12-31 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Oculopharyngeal Muscular Dystrophy
Interventions
- Genetic: BB-301: Dose escalation phase 1b cohort 1
- Genetic: BB-301: Dose escalation phase 1b cohort 2
- Genetic: BB-301: Dose escalation phase 1b cohort 3
- Genetic: BB-301: Dose expansion phase 2a
Primary outcomes
what the primary-completion date above is the date OFIncidence of dose-limiting toxicities (DLTs) in phase 1b
measured Up to 60 days
Incidence of adverse events (AEs) according to NCI CTCAE v5.0 in phase 1b and in phase 2a
measured Up to 360 days
Phase 1b: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 1b: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2
measured 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)
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2
measured Baseline, Day 90, Day 180, Day 270, Day 360
Phase 2a: Pharyngeal constrictor muscle function as estimated by PhAMPC
measured Baseline, Day 90, Day 180, Day 270, Day 360
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