Hearing loss is the most common sensory disability worldwide, affecting one in five people across all ages. While hearing aids and cochlear implants help many, they don't restore natural hearing — and for congenital genetic deafness, new approaches offer the possibility of genuine biological hearing for the first time.
What's actually going on in research
Gene therapy for OTOF (otoferlin) mutations — the most common cause of severe congenital hearing loss — has produced dramatic hearing restoration in early clinical trials, with children going from profound deafness to hearing speech. Regenerative approaches targeting hair cell regrowth via Atoh1 gene delivery and Notch pathway inhibition are in early clinical testing. Ototoxicity protection drugs are in trials to prevent hearing loss caused by chemotherapy, particularly in children.
Gene therapy for deafness
Delivering functional OTOF and other deafness-gene replacements via viral vectors into the inner ear has enabled children born deaf to hear speech. Trials are expanding to more genetic causes.
Hair cell regeneration
Drugs activating the Atoh1 transcription factor or inhibiting Notch signaling can stimulate supporting cells in the cochlea to become new hair cells in animal models, and early human trials are beginning.
Ototoxicity prevention
Sodium thiosulfate and other drugs are being tested to protect the inner ear hair cells from cisplatin chemotherapy-induced damage in children with cancer.
What to know before you search
Eligibility depends on the cause and degree of hearing loss, specific genetic mutation identified, age at intervention, and whether there is residual hearing.
What types of trials are currently open
- Gene therapy trials — Testing inner ear gene delivery for specific genetic causes of hearing loss including OTOF mutations.
- Regenerative trials — Evaluating drugs and gene approaches to stimulate cochlear hair cell regrowth.
- Device trials — Comparing cochlear implant designs, electrode arrays, and programming strategies.
- Ototoxicity prevention trials — Testing agents to protect hearing during chemotherapy or antibiotic treatment.
- Hearing rehabilitation trials — Evaluating hearing aid technology, auditory training programs, and tinnitus management.
Recently added Hearing Loss trials
A Randomized Trial of Intratympanic Drug Delivery: Evaluating the Efficacy and Safety of Dexamethasone-Loaded Exosomes Versus Standard Therapy in Acute Sensorineural Hearing Loss.
Primary Objective To compare the efficacy of a single course of IT Exo-Dex versus conventional IT dexamethasone and exosome vehicle alone, as measured by the mean change in pure-tone average (PTA; 0.5, 1, 2, 4 kHz) from baseline to the 4-week post-treatment endpoint. Secondary Objectives 1. To determine the safety and tolerability profile of IT Exo-Dex. 2. To compare the rate of hearing recovery (defined as \>10 dB improvement in PTA or recovery to within 10 dB of contralateral ear) among the three treatment groups at 1, 4, and 12 weeks. 3. To assess changes in auditory function via Auditory Brainstem Response (ABR) thresholds and Otoacoustic Emissions (OAEs). 4. To characterize the pharmacokinetics and inner ear biodistribution of Exo-Dex using advanced imaging modalities (e.g., MRI with exosome-contrast agents in a sub-study cohort if applicable).
Wideband Tympanometry as a Non-behavioral Test of the Speech-weighted Middle Ear Transfer Function.
There is a need for a non-behavioral clinical test that provides an assessment of how speech is conducted through the middle ear for the optimization of middle ear implants. These implants utilize the functionality of the human middle ear and require adequate speech conduction through the middle ear chain. Consequently, the clinical test of this degree of speech transfer can contribute to the pre-operative indication for middle ear implants. Wideband Tympanometry (WT) is a non-invasive diagnostic tool for the clinical assessment of the condition of the middle ear. Unlike classical tympanometry, WT tests the impedance of the middle ear system as a function of both pressure and frequencies. Consequently, a WT measurement contains many times more information about the functionality of the middle ear than classical tympanometry and is ideally suited as a diagnostic tool for the clinical assessment of speech transfer through the middle ear. The WT measurements will be correlated with the Speech Intelligibility Index (SII) collected in normally hearing individuals and patients with isolated conductive hearing loss. This index is a measure of speech transfer through the middle ear. Consequently, a correlation of the WT measurements with the SII addresses the need for a non-behavioral clinical test of speech transfer through the middle ear. Additionally, WT measurements will be performed longitudinally in cochlear implant candidates, one of the populations that can benefit from the developed clinical test. These longitudinal WT measurements, which will be performed both pre- and post-operatively, allow for the evaluation of the impact of middle ear surgery on the predicted SII.
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