Actively Recruiting
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Led by Benitec Biopharma, Inc. · Updated on 2025-12-31
30
Participants Needed
1
Research Sites
883 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Previously enrolled in the BNTC-OPMD-NH-001 natural history study and completed at least 6 months of follow-up
- Signed written informed consent before any study procedures
- Male or female aged 50 to 65 years at natural history study enrollment with genetically diagnosed heterozygous or homozygous OPMD
- Eligible and willing to undergo surgical injection of the drug into pharyngeal muscles under general anesthesia
- Moderate dysphagia defined by specific swallowing muscle function measurements
- Not of childbearing potential (postmenopausal or surgical sterilization) or agrees to use two highly effective contraceptive methods during and 52 weeks after the study
- Subjects able to abstain from heterosexual intercourse or use backup contraception if sexually active during the study
- Subjects capable of donating sperm agree not to do so from screening through 52 weeks after drug administration
You will not qualify if you...
- Prior treatment with any adeno-associated virus (AAV) vector therapy
- Presence of anti-AAV9 antibodies above 1:50 titer
- Pregnancy or breastfeeding
- Pharyngeal muscle not suitable for injection due to significant muscle atrophy
- Contraindications to videofluoroscopy or allergy to contrast agents
- Gene therapy treatment within 6 months before screening
- Contraindications to study procedures or medications like corticosteroids
- Prior cricopharyngeal myotomy or botulinum toxin injection
- Cricopharyngeal dilation within 12 months prior to screening
- Hospitalization for pneumonia related to nonpathogenic causes or bacterial infection within 12 months prior to screening
- Intubation within 30 days prior to screening
- Very restricted diet texture score of 3 or less
- Other neuromuscular diseases or disorders that affect swallowing
- Other disorders causing dysphagia such as severe reflux or esophageal conditions
- Serious illnesses or cancers not in remission for at least 5 years
- History of head and neck cancer or neck radiation
- Major mouth or neck surgery except routine dental, tonsillectomy, or adenoidectomy
- Abnormal liver function tests
- Immunocompromised or on immunosuppressants
- Significant hematological, renal, endocrine, lung, gastrointestinal, heart, psychiatric, neurological, or allergic conditions
- Severe hypertension as defined by specific blood pressure criteria
- Malnutrition or cachexia based on weight loss and body mass index
- Recent treatment with investigational drugs or devices
- Conditions compromising ability to consent or comply with study
- Any other medical or social condition preventing study completion or informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
Research Team
J
Jerel A. Banks, M.D., Ph.D.
CONTACT
S
Sophie Mukadam
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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