New York, NYNCT06185673Now EnrollingIRB Ready

Oculopharyngeal Muscular Dystrophy Clinical Trial in New York, NY

Access cutting-edge oculopharyngeal muscular dystrophy treatment through this phase 1/2 clinical trial at a research site in New York. Study-provided care at no cost to qualified participants.

Sponsored by Benitec Biopharma, Inc.

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Expert Care in New York

Access oculopharyngeal muscular dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related oculopharyngeal muscular dystrophy treatment provided free

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Check if you qualify for this oculopharyngeal muscular dystrophy clinical trial in New York, NY

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Why Participate?

  • No-Cost Study Care

  • Local to New York

    Convenient for NY residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit New York site if eligible
  4. 4Begin participation

About This Oculopharyngeal Muscular Dystrophy Study in New York

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.

Sponsor: Benitec Biopharma, Inc.

Who Can Participate

Inclusion Criteria

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 Criteria

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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in New York?

Yes, this clinical trial (NCT06185673) has an active research site in New York, NY that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Oculopharyngeal Muscular Dystrophy Treatment Options in New York, NY

If you're searching for oculopharyngeal muscular dystrophy treatment options in New York, NY, this clinical trial (NCT06185673) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our New York research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced oculopharyngeal muscular dystrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all oculopharyngeal muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

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