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
Hidden Hearing Loss in Older Adults
The goal of this observational study is to investigate whether speech-in-noise perception deficits in older adults aged 60 years or older with objectively normal hearing thresholds are due to impaired ability of processing temporal cues in speech signals. The study further aims to examine whether cognitive and cortical mechanisms provide compensatory support for speech perception in challenging listening environments.
First Clinical Study to Evaluate Safety, Tolerability & PK of DX243 in Healthy Volunteers and Patients With Hearing Loss
Phase 2a: Multiple Ascending Dose (MAD) in male and female patients with hearing loss: The study will be conducted according to a randomised, placebo-controlled, double-blind design. A total of 24 patients, otherwise healthy, aged up to 75 years old, with mild to moderate hearing loss will be included. Two cohorts of 12 male or female patients (no ratio is required) will receive two different flat doses (low dose and high dose) ofDX243 or placebo for 29 days using SC administration. In each cohort of 12 patients, 4 will be randomised to placebo and 8 to DX243, so at the end of Phase 2a, 8 patients will have received placebo, 8 the low dose and 8 the high dose of DX243. The primary objective is to evaluate the safety and tolerability of DX243 administered subcutaneously after repeated doses. The secondary objectives are to detect preliminary signal of efficacy, using speech in noise tests, tonal and vocal audiometry, as well as tinnitus and quality of life.
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