Phase 2 Muscular Dystrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who were previously enrolled in a related natural history study called BNTC-OPMD-NH-001 and completed at least 6 months of follow-up in that study.
• People who gave written consent before any study procedures began.
• People aged 50 to 65 years at the time of enrolling in the natural history study, who have a genetic diagnosis of OPMD with one changed copy of the relevant gene (heterozygous), with specific gene repeat patterns: GCN10/GCN12, GCN10/GCN13, GCN10/GCN14, GCN10/GCN15, or GCN10/GCN16.
• People aged 65 years or under at the time of enrolling in the natural history study, who have a genetic diagnosis of OPMD with two changed copies of the relevant gene (homozygous), with specific gene repeat patterns: GCN12/GCN12, GCN13/GCN13, GCN14/GCN14, GCN15/GCN15, or GCN16/GCN16.
• People who are willing and medically suitable to undergo a surgical procedure to the throat area under general anaesthesia to receive the study drug.
• People who have a moderate level of difficulty swallowing, as measured by specific results from a swallowing X-ray test (videofluoroscopy) meeting defined thresholds (confirm with trial site).
• People who are not able to become pregnant — for example, those who have gone through menopause (no menstrual period for at least 1 year with no other medical cause), or who have had a hysterectomy, removal of both fallopian tubes, or removal of both ovaries.
• People who are able to become pregnant, or whose partner is able to become pregnant, and who agree to use two highly effective forms of contraception during the study and for 52 weeks after receiving the study drug — specifically, a condom used together with one of the following: bilateral tubal ligation; hormonal contraception (such as the pill, vaginal ring, implant, or injection) that has been at a stable dose for at least 1 month before dosing; or an intrauterine device (IUD) inserted at least 1 month before dosing.
• People who agree to abstain completely from heterosexual intercourse for the full duration of the study and for 52 weeks after receiving the study drug, and also agree to use a highly effective contraceptive method as a backup if they become sexually active — this is only acceptable if complete abstinence is already their usual lifestyle (periodic or calendar-based abstinence, withdrawal, and spermicides alone are not accepted).
• People who are able to donate sperm must agree not to do so from the screening visit through 52 weeks after receiving the study drug.

Who may not be able to join:

• People who have previously received any gene therapy using an adeno-associated virus (AAV) vector — for example, treatments for haemophilia B (such as HEMGENIX®) or for an inherited eye condition caused by RPE65 gene mutations (such as LUXTURNA®).
• People who have a high level of antibodies in their blood against the AAV9 virus (a titre above 1:50), which could affect how the body responds to the study drug (confirm with trial site).
• People who are currently pregnant or breastfeeding.
• People whose throat muscles are judged by the study doctor to be too weakened or wasted to safely receive an injection into the muscle, based on swallowing X-ray measurements compared to healthy volunteers (confirm with trial site).
• People who cannot undergo the swallowing X-ray procedure — for example, due to an allergy to the contrast agent (a liquid used to make the X-ray images visible).
• People who have received any other type of gene therapy — including CAR-T cell therapies (such as ABECMA®, BREYANZI®, CARVYKTI™, KYMRIAH®, YESCARTA®, or TECARTUS™), IMLYGIC®, SKYSONA®, or ZYNTEGLO® — within the 6 months before the screening visit.
• People for whom any of the study procedures or medications used in the trial (such as corticosteroids/steroid medicines) would be medically unsuitable or harmful.
• People who have previously had a cricopharyngeal myotomy (a specific throat muscle surgery) or botulinum toxin (Botox) injections into the cricopharyngeal muscle in the throat.
• People who have had a cricopharyngeal dilation procedure (a stretching procedure of the throat) within the 12 months before the screening visit.
• People who have been hospitalised in the 12 months before the screening visit for pneumonia that was not caused by an identifiable infectious organism (such as aspiration pneumonitis or bacterial pneumonia) — though hospitalisation for pneumonia caused by COVID-19, other viruses, or fungal infections does not automatically exclude someone (confirm with trial site).
• People who have been put on a breathing tube (intubated) within the 30 days before the screening visit.
• People who are eating a very limited range of food textures, defined as a score of 3 or below on the IDDSI Functional Diet Scale as assessed during the natural history study screening visit (confirm with trial site).
• People who have another muscle-wasting or neuromuscular disease in addition to OPMD, or any other condition that could make it hard to accurately assess swallowing difficulties related to OPMD.
• People who have other conditions known to cause swallowing difficulties — such as severe acid reflux, narrowing of the oesophagus (food pipe) from injury or infection, swallowing problems caused by certain medications (such as bisphosphonates), or spastic movement disorders of the oesophagus.
• People with a serious illness that is likely to significantly shorten life expectancy, or with any cancer other than certain treated skin cancers or cervical carcinoma in situ, unless the cancer has been fully treated or has been in complete remission for 5 years or more.
• People who have ever been diagnosed with head or neck cancer.
• People who have had radiation treatment to the neck at any time in the past.
• People who have had major surgery to the mouth or neck — though a history of routine dental work, tonsil removal, or adenoid removal does not exclude someone.
• People with abnormal liver function, specifically blood test results showing liver enzymes (ALT or AST) more than 2.5 times the normal upper limit.
• People who have a weakened immune system or who are currently taking medicines that suppress the immune system.
• People with significant ongoing medical conditions affecting the blood, kidneys, hormones, lungs, digestive system, heart, mental health, nervous system, or who have clinically significant allergies (including drug allergies).
• People with severely high blood pressure, defined as a consistently recorded systolic pressure of 160 mmHg or above, or diastolic pressure of 100 mmHg or above.
• People with malnutrition, defined as unintended weight loss of more than 5–10% over 1 to 6 months combined with a body mass index (BMI) below 18.5–20 kg/m², or with cachexia, defined as weight loss of more than 5% over 6 months or a BMI below 20 kg/m² with ongoing weight loss of more than 2%.
• People who have taken part in another clinical trial or used an investigational drug or device within the 3 months (or 5 half-lives of the drug) before the screening visit.
• People who, in the study doctor's opinion, have a condition that affects their ability to give informed consent or to follow the study procedures.
• People who are unwilling or unable to follow study procedures and attend all required follow-up visits.
• People who have any other medical or personal circumstance that, in the study doctor's opinion, would prevent them from completing the study or giving informed consent.

Important: Always verify eligibility with the trial site directly before applying.