Phase 2 Hearing Loss 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:

• The participant (and/or their legal guardian) must be willing to give written consent to take part, attend all follow-up visits, and understand what the trial involves and what results may or may not be possible
• The participant must be able to communicate with the research team and follow the study instructions, with guardian support if needed
• Children aged 1 to 17 years old (male or female) at the time of joining the trial
• Genetic testing must show a specific type of inherited deafness called DFNB9, caused by changes in both copies of the Otoferlin gene
• Hearing tests (ABR) must show severe or profound hearing loss of 65 dB or greater; the first participant enrolled must have a result above 90 dB
• Ear scans (CT and/or MRI taken within 3 months) must confirm the ear is suitable for surgery, with no structural problems, nerve abnormalities, or active ear infection, and the surgeon must consider the participant suitable for the procedure
• A specific hearing test called DPOAE must show a detectable response

Who may not be able to join:

• People whose hearing loss is caused by structural problems in the middle or inner ear, nerve abnormalities, sound-conducting issues, mixed hearing loss, or deafness linked to other physical abnormalities
• People with existing ear conditions that the investigator believes could affect the surgery or trial results, such as ear infections, Ménière's disease, acoustic neuroma, or unresolved sudden hearing loss
• People with a history of drug abuse
• People who have taken certain medications known to damage hearing (such as some antibiotics, a chemotherapy drug called cisplatin, or certain water tablets) within the past 6 months
• People who have received antiviral or immune-based treatments within the 3 months before screening
• People who have received a live vaccine within 30 days before screening
• People with a weakened immune system or immune disorders, including a positive HIV test, any congenital or acquired immune deficiency, or a history of organ transplantation
• People with serious ongoing illnesses or active conditions such as tuberculosis, herpes zoster, pancreatitis, kidney problems, or stomach/intestinal ulcers
• People with conditions that make surgery or anaesthesia unsafe, including heart or stroke-related events in the past 6 months, other heart conditions the investigator considers a concern, or a known allergy to the study drug
• People who have taken part in a gene therapy trial within the past 6 months, plan to join another interventional trial within one year after treatment, or have recently received another experimental drug (within a timeframe based on how long that drug stays in the body)
• People who already have a cochlear implant or similar device in the ear being treated
• People with other serious conditions present from birth
• People with a history of neurological or psychiatric conditions such as epilepsy or dementia
• People who take long-term blood-thinning medication that cannot be safely paused
• People who have had radiotherapy or chemotherapy that the investigator believes could affect the trial results
• People who test positive for hepatitis B, hepatitis C (with active virus detected), HIV, or active syphilis
• Girls or women who could become pregnant and test positive for pregnancy before the dose is given; and girls or women of childbearing potential, or males who have not been sterilised and have a fertile partner, who are unwilling or unable to use effective contraception from the time of consent until at least 24 months after the dose
• Anyone the investigator considers unsuitable for any other reason (confirm with trial site)

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