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A Phase 1b/2a, Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

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Trial Profile

A Phase 1b/2a, Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 24 Mar 2025

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At a glance

  • Drugs BB 301 (Primary)
  • Indications Oculopharyngeal muscular dystrophy
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Benitec Biopharma

    • Most Recent Events

    • 19 Mar 2025 According to a Benitec Biopharma media release, an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, taking place in Dallas, Texas, on March 19, 2025 at 1:15 pm Central Time .
    • 19 Mar 2025 Interim results presented in the Benitec Biopharma Media Release.
    • 14 Feb 2025 According to a Benitec Biopharma media release, the company announces The fourth Subject was safely treated with the low-dose of BB-301 in December 2024, and the fifth Subject was safely treated with the low-dose of BB-301 in early February 2025. The sixth Subject is expected to be treated with the low-dose of BB-301 in 2Q 2025 and company expects to begin treating a second cohort of OPMD Subjects with a higher dose of BB-301 later in the year.

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