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BB-301 for Oculopharyngeal Muscular Dystrophy with Dysphagia

This study is testing a treatment called BB-301 for people with Oculopharyngeal Muscular Dystrophy (OPMD) who experience difficulty swallowing (dysphagia). BB-301 is a gene therapy that aims to deliver a healthy PABPN1 protein and reduce the faulty protein causing OPMD. It will be injected directly into the throat muscles. The study is looking at how safe BB-301 is, what the best dose is, and how well it improves swallowing. To join, you must have previously participated in a specific OPMD natural history study (BNTC-OPMD-NH-001) for at least 6 months, be between 50 and 65 years old, and have a genetic diagnosis of OPMD. The study plans to enroll 30 participants, but its current status is unclear.

Study design
This is an interventional study, meaning participants will receive a treatment. It involves a dose escalation phase (Phase 1b) to find the right dose, followed by a dose expansion phase (Phase 2a).
What's involved
You would receive a single dose of BB-301 injected into your throat muscles during an open surgical procedure under general anesthesia. You would have follow-up visits for up to 360 days to monitor safety and swallowing efficiency.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety for up to 360 days. Swallowing efficiency will be measured at baseline, 90, 180, 270, and 360 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06185673

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

Recruiting
PHASE1Up to 65InterventionalTreatment
Benitec Biopharma, Inc.
~30 participants
Updated 2025-12-31 on ClinicalTrials.gov
What's tested:BB-301: Dose escalation phase 1b cohort 1BB-301: Dose escalation phase 1b cohort 2BB-301: Dose escalation phase 1b cohort 3BB-301: Dose expansion phase 2a

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose-limiting toxicities (DLTs) in phase 1b
Measured over Up to 60 days
+11 more outcomes measured

Conditions

Oculopharyngeal Muscular Dystrophy

Explore related trials

Oculopharyngeal Muscular DystrophyPABPN1 Gene

NCT06185673

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • NYU Langone Health

    New York, New Yorkstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Milan R. Amin, M.D. · PRINCIPAL_INVESTIGATOR · NYU Langone Health

Who to contact

Jerel A. Banks, M.D., Ph.D.
[email protected]
(510)-780-0634
Email the study team

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Eligibility criteria

Inclusion

Subject was previously enrolled in the BNTC-OPMD-NH-001 natural history (NH) study and completed at least 6 months of follow-up in the NH study.
Signed written informed consent prior to the initiation of any study-specific procedures.
Males or females, aged ≥50 to ≤65 years at the time of NH study enrollment, with genetically diagnosed heterozygous OPMD disease (as indicated by 1 of the following allelic classifications: GCN10/GCN12, GCN10/GCN13, GCN10/GCN14, GCN10/GCN15, GCN10/GCN16) OR
Males or females, aged ≤65 years at the time of NH study enrollment, with genetically diagnosed homozygous OPMD disease (as indicated by 1 of the following allelic classifications: GCN12/GCN12, GCN13/GCN13, GCN14/GCN14, GCN15/GCN15, GCN16/GCN16).
Subject is eligible and willing to undergo a surgical dissection of the pharyngeal region with intubation under general anesthesia to administer the study drug.
Subject has moderate dysphagia, defined as pharyngeal area at maximum constriction (PhAMPC) \>2.7%(C2-4)\^2 with natural sips of thin liquid barium or PhAMPC \>2.1%(C2-4)\^2 with teaspoon delivery of moderately thick liquid barium.
Subject is not of childbearing potential, i.e., is postmenopausal (absence of menstrual bleeding for ≥1 year before Baseline, without any other medical reason), or has documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy OR
Subject or their partner is of childbearing potential and agrees to use 2 highly effective forms of contraception during the study and continuing through 52 weeks after the study drug administration. The 2 authorized forms of contraception are condom used with 1 of the following methods of contraception:
bilateral tubal ligation
combined oral contraceptives (estrogens and progesterone), vaginal ring, or implanted or injectable hormonal contraceptives with a stable dose for at least 1 month prior to the day of dosing; hormonal contraceptives must inhibit ovulation
intrauterine device inserted at least 1 month prior to the day of dosing OR
Subject agrees to abstain from heterosexual intercourse during study participation and to use a highly effective form of contraception (as described above) as backup if they become sexually active during the study. Abstinence is only acceptable if this is the subject's usual lifestyle. Periodic abstinence (calendar, symptothermal, postovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception.
Subjects capable of donating sperm must agree not to donate sperm beginning at Screening and continuing through 52 weeks after the study drug administration.

Exclusion

Subject has received prior treatment with an adeno-associated virus (AAV) vector (e.g., AAV-based therapy for the treatment of hemophilia B \[including HEMGENIX®\], AAV-based therapy for the treatment of RPE65 mutation-associated retinal dystrophy \[including LUXTURNA®\]).
Subject with presence of anti-AAV9 antibody titers \>1:50.
Subject is pregnant or breastfeeding.
In the investigator's opinion, the subject's pharyngeal muscle is not amenable to intramuscular (IM) injection due to clinically significant atrophy as assessed by maximum pharyngeal dilation for OPMD subjects (determined by normalized post-swallow hyoid rest pharyngeal area \[HRAN\] using videofluoroscopy) compared to relative HRAN measurements of pharyngeal dilation from a database comprising healthy control subjects as determined during the Screening Visit of the NH study.
Subject with contraindication to the videofluoroscopy procedures (e.g., allergy to any of the radiopaque contrast agents planned for use in the study).
Subject has received gene therapy (e.g., chimeric antigen receptor-positive T cell therapy for the treatment of leukemia, lymphoma, or multiple myeloma \[including ABECMA®, BREYANZI®, CARVYKTI™, KYMRIAH®, YESCARTA®, and TECARTUS™\], IMLYGIC® for the treatment of melanoma, SKYSONA® for the treatment of cerebral adrenoleukodystrophy, and ZYNTEGLO® for the treatment of β-thalassemia) within the 6 months prior to Screening.
Subject for whom any of the proposed study procedures or medications (e.g., corticosteroids) would be contraindicated.
Subject has had prior cricopharyngeal myotomy or cricopharyngeal botulinum toxin injection.
Subject has had cricopharyngeal dilation within the 12 months prior to Screening.
Subject with pre-existing clinically diagnosed and/or self-reported dysphagia has been hospitalized within the 12 months prior to Screening for treatment of pneumonia of nonpathogenic origin (e.g., aspiration pneumonitis secondary to aspiration of sterile gastric contents) or pneumonia secondary to bacterial pathogens. A subject is eligible for enrollment if diagnosed with pneumonia secondary to documented pathogenic organism(s)including: severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2, i.e., coronavirus disease 2019), non-SARS-CoV-2-related viral pathogens, and fungal pathogens.
Subject has been intubated within the 30 days prior to Screening.
Subject consumes a very restricted range of diet textures, defined as a score of ≤3 on the IDDSI Functional Diet Scale as determined during the Screening Visit of the NH study.
Subject presents with muscular dystrophy and/or other neuromuscular diseases distinct from OPMD, or any other disease that may significantly interfere with the characterization of dysphagia in OPMD.
Subject presents with other disorders associated with dysphagia, e.g., severe gastroesophageal reflux, esophageal stricture due to mechanical or chemical trauma, infection (e.g., esophageal moniliasis), drug-induced dysphagia (e.g., bisphosphonates), esophageal rings and webs, or spastic motility disorders of the esophagus.
Subject with any concomitant illness likely to significantly decrease life expectancy or any malignancy other than curatively treated skin cancer or in situ carcinoma of the cervix, unless adequately treated or in complete remission for ≥5 years.
Subject has received a diagnosis of head and neck cancer at any time.
Subject has any history of neck irradiation.
Subject has undergone a major surgical procedure to the mouth or neck. Subject is eligible for enrollment with a past medical history of routine dental procedures, tonsillectomy, or adenoidectomy.
Subject has abnormal liver function (serum alanine aminotransferase or aspartate aminotransferase \>2.5 × upper limit of normal).
Subject is immunocompromised or is receiving immunosuppressant therapy.
Subject has evidence of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, psychiatric, neurologic, or allergic diseases (including drug allergies but excluding untreated, not clinically significant, seasonal allergies).
Subject has NCI CTCAE grade 3 hypertension defined as systolic blood pressure consistently ≥160 mmHg or diastolic blood pressure consistently ≥100 mmHg.
Subject has malnutrition defined as unintended weight loss of \>5-10% during the 1 to 6 months prior to Screening and a body mass index (BMI) of \<18.5 to 20 kg/m², or subject has cachexia defined as weight loss of \>5% over the past 6 months or a BMI of \<20 kg/m² and ongoing weight loss of \>2%.
Subject has received treatment with an investigational drug, investigational device, or approved therapy for investigational use within the 3 months or 5 half-lives prior to Screening.
Subject has any kind of disorder that, in the investigator's opinion, compromises the ability of the subject to give written informed consent and/or to comply with study procedures.
Subject is unwilling or unable to comply with study procedures and scheduled follow-up visits.
Subject has any other medical or social condition that, in the investigator's opinion, would not permit the subject to complete the study or sign informed consent.

What this trial measures

  • Incidence of dose-limiting toxicities (DLTs) in phase 1bUp to 60 days

    A DLT will be defined using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0, as follows: • Any Grade 2 toxicity not resolving within 14 days or any Grade 3 toxicity, assessed to be possibly related to the investigational product.

  • Incidence of adverse events (AEs) according to NCI CTCAE v5.0 in phase 1b and in phase 2aUp to 360 days

    For this outcome measure, AEs arising in the 360 days following administration of BB-301 will be considered. Long term AEs will be monitored for 15 years following subject dosing.

  • Phase 1b: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The Analysis of Swallowing Physiology: Events, Kinematics and Timing (ASPEKT) method will be used to determine Vallecular Residue %(C2-4)\^2.

  • Phase 1b: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Pyriform Sinus Residue %(C2-4)\^2.

  • Phase 1b: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Other Pharyngeal Residue %(C2-4)\^2.

  • Phase 1b: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Total Pharyngeal Residue %(C2-4)\^2.

  • Phase 1b: Pharyngeal constrictor muscle function as estimated by the pharyngeal area at maximum constriction (PhAMPC)Baseline, Day 90, Day 180, Day 270, Day 360

    Videofluoroscopy will be used to characterize the area of the pharynx at the point of maximum constriction during swallowing. The PhAMPC uses the videofluoroscopy frame of maximum pharyngeal constriction, defined as the frame with the smallest amount of unobliterated air space and barium-containing bolus visible in the pharynx. The pixelated area of the frame of maximum constriction is normalized via the use of the C2-C4 length squared (i.e., \[C2-4\]\^2) as the denominator.

  • Phase 2a: Swallowing efficiency as measured by Vallecular Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Vallecular Residue %(C2-4)\^2.

  • Phase 2a: Swallowing efficiency as measured by Pyriform Sinus Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Pyriform Sinus Residue %(C2-4)\^2.

  • Phase 2a: Swallowing efficiency as measured by Other Pharyngeal Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Other Pharyngeal Residue %(C2-4)\^2.

  • Phase 2a: Swallowing efficiency as measured by Total Pharyngeal Residue %(C2-4)^2Baseline, Day 90, Day 180, Day 270, Day 360

    Pharyngeal residue in discrete anatomical locations will be assessed by applying a method that uses a common reference area across all residue locations, with residue area expressed as a percentage of the squared C2-C4 length reference scalar. The ASPEKT method will be used to determine Total Pharyngeal Residue %(C2-4)\^2.

  • Phase 2a: Pharyngeal constrictor muscle function as estimated by PhAMPCBaseline, Day 90, Day 180, Day 270, Day 360

    Videofluoroscopy will be used to characterize the area of the pharynx at the point of maximum constriction during swallowing. The PhAMPC uses the videofluoroscopy frame of maximum pharyngeal constriction, defined as the frame with the smallest amount of unobliterated air space and barium-containing bolus visible in the pharynx. The pixelated area of the frame of maximum constriction is normalized via the use of the C2-C4 length squared (i.e., \[C2-4\]\^2) as the denominator.