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Genetic Therapy

BB-301 Treatment for Oculopharyngeal Muscular Dystrophy

Phase 1 & 2

Recruiting

Led By Milan R. Amin, M.D.

Research Sponsored by Benitec Biopharma, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Males or females, aged ≥50 to ≤65 years at the time of NH study enrollment, with genetically diagnosed heterozygous OPMD disease or aged ≤65 years at the time of NH study enrollment, with genetically diagnosed homozygous OPMD disease.

Subject has moderate dysphagia, defined as specific pharyngeal area measurements with natural sips of thin liquid barium or teaspoon delivery of moderately thick liquid barium.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, day 90, day 180, day 270, day 360

Awards & highlights

Study Summary

This trial is for people with a condition called oculopharyngeal muscular dystrophy (OPMD). Participants who have been in another study for at least 6 months may be able to take

Who is the study for?

This trial is for individuals with Oculopharyngeal Muscular Dystrophy (OPMD) who have trouble swallowing (dysphagia) and have been part of a previous OPMD study for at least 6 months. Participants will undergo surgery under general anesthesia to receive the treatment.Check my eligibility

What is being tested?

The trial tests different doses of BB-301, an investigational drug, injected into throat muscles during surgery. The goal is to find the safest and most effective dose to improve swallowing in OPMD patients.See study design

What are the potential side effects?

Potential side effects may include reactions related to the surgical procedure, such as pain or infection at the injection site, complications from anesthesia, and any unforeseen effects of BB-301.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 50 and 65 years old with a genetic diagnosis of OPMD.

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I have moderate difficulty swallowing.

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I am willing and able to have surgery in my throat area under general anesthesia for the study drug.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, day 90, day 180, day 270, day 360

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, day 90, day 180, day 270, day 360

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, day 90, day 180, day 270, day 360 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of adverse events (AEs) according to NCI CTCAE v5.0 in phase 1b and in phase 2a

Incidence of dose-limiting toxicities (DLTs) in phase 1b

Phase 1b: Pharyngeal constrictor muscle function as estimated by the pharyngeal area at maximum constriction (PhAMPC)

+9 more

Secondary outcome measures

Phase 1b: Dysphagia severity as measured by the cold water timed drinking test (CWDT)

Phase 1b: Global swallowing function as measured by the Dynamic Imaging Grade of Swallowing Toxicity (DIGEST) scale

Phase 1b: Maximum pharyngeal dilation as estimated by %(C2-4)^2-normalized post-swallow hyoid rest pharyngeal area (HRAN)

+15 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: BB-301 TreatmentExperimental Treatment4 Interventions

The phase 1b component of the study is the dose escalation phase which will enroll up to 18 subjects in up to 3 dosing cohorts. The phase 2a component of the study is the dose expansion phase which will enroll up to 12 subjects.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Benitec Biopharma, Inc.Lead Sponsor

1 Previous Clinical Trials

16 Total Patients Enrolled

Milan R. Amin, M.D.Principal InvestigatorNYU Langone Health

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the upper limit for the total number of individuals involved in this medical study?

"Indeed, as stated on clinicaltrials.gov, this trial is presently seeking eligible participants. The study was initially posted on November 28th, 2023 and has since been updated most recently on December 15th, 2023. A total of 30 patients are being sought for enrollment at a single location."

Answered by AI

Are individuals currently able to actively enroll in this research study?

"Indeed, according to the details provided on clinicaltrials.gov, this trial is currently seeking eligible participants. The initial posting of the study occurred on November 28th, 2023 and it was most recently updated on December 15th, 2023."

Answered by AI

What are the main goals being pursued in this research endeavor?

"Secondary outcomes include evaluating dysphagia severity using the cold water timed drinking test (CWDT) which measures the time in seconds it takes for a subject to consume 80 mL of ice-cold water. Additionally, global swallowing function will be assessed using the Dynamic Imaging Grade of Swallowing Toxicity ("

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

2023. "A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06185673.

All > Dysphagia > Muscular Dystrophy