Ongoing Clinical Trials for Deafness
This article provides an overview of 5 ongoing clinical trials investigating potential treatments for deafness and hearing loss. These studies focus on gene therapies for genetic forms of hearing loss caused by mutations in the otoferlin gene, protective agents against chemotherapy-induced hearing damage, and treatments for Meniere’s disease. Trials are currently taking place in Spain, Germany, the Netherlands, and France.
Clinical trial locations
- France
- Germany
- Netherlands
- Spain
- Study on DB-OTO for Safety and Tolerability in Infants and Children with Genetic Hearing Loss
- Study on Gene Therapy AAVAnc80-hOTOF for Patients with Sensorineural Hearing Loss from Otoferlin Gene Mutations
- Study on Long-Term Safety of AAVAnc80-hOTOF Gene Therapy for Patients with Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
Study on DB-OTO for Safety and Tolerability in Infants and Children with Genetic Hearing Loss
This trial is investigating a gene therapy called DB-OTO for children and infants with congenital hearing loss caused by mutations in both copies of the otoferlin gene. The otoferlin gene is essential for proper functioning of the inner ear’s hair cells, which convert sound into nerve signals.
Who can participate: Children under 18 years of age with confirmed mutations in both copies of the OTOF gene are eligible. For infants up to 24 months, there must be no response in auditory brainstem response testing at certain sound levels, but otoacoustic emissions must be present in the ear to be treated. For older children, similar hearing test requirements apply, with either otoacoustic emissions or cochlear microphonic present. Parents or legal guardians must consent to vaccination according to the national schedule, and participants must not have hearing loss that changes with body temperature.
Who cannot participate: Children who do not have congenital hearing loss caused by mutations in both copies of the otoferlin gene are excluded. Those outside the specified age range, with other medical conditions that might interfere with the study, or who cannot follow study procedures are also ineligible. Recent participation in another clinical trial or allergies to the study medication may also prevent participation.
What the trial involves: DB-OTO is administered as an injection directly into the inner ear. The study begins with treatment in one ear and may later include both ears if well-tolerated. Participants are closely monitored for side effects and undergo regular hearing tests, including auditory brainstem response testing and behavioral audiometry, to assess changes in hearing ability. The goal is to determine the safest and most effective dose while evaluating how well the treatment is tolerated.
Investigational treatment: DB-OTO consists of two components that work together to deliver a functional copy of the otoferlin gene to the inner ear, potentially helping restore normal hearing function in affected children.
Study on Long-Term Safety of AAVAnc80-hOTOF Gene Therapy for Patients with Sensorineural Hearing Loss Due to Otoferlin Gene Mutations
This follow-up study evaluates the long-term safety of AAVAnc80-hOTOF gene therapy in patients who previously received this treatment in an earlier clinical trial. The therapy uses a special delivery system to introduce a normal version of the otoferlin gene into the inner ear.
Who can participate: Individuals of any age or gender who have otoferlin gene-mediated hearing loss and have already received intracochlear administration of AAVAnc80-hOTOF in a previous clinical trial are eligible. The participant or their legally authorized representative must agree to follow all study requirements and complete the informed consent process.
Who cannot participate: Those who do not have otoferlin gene-mediated hearing loss or who were not treated with AAVAnc80-hOTOF in a previous trial cannot participate. Certain vulnerable populations may be excluded depending on specific study provisions.
What the trial involves: This is a long-term monitoring study that tracks participants over an extended period, with the study expected to continue until 2033. Regular follow-up visits include physical examinations, laboratory tests including blood and urine analysis, and comprehensive hearing evaluations. Safety assessments focus on identifying any late-occurring adverse events, changes in vital signs, and laboratory test results. Hearing assessments include auditory brainstem response testing, behavioral audiometry, and speech recognition tests.
Investigational treatment: AAVAnc80-hOTOF was previously administered as a single injection into the inner ear, delivering a healthy copy of the otoferlin gene to help restore hearing function.
Study on Meniere’s Disease: Comparing Methylprednisolone and Placebo for Patients with Unilateral Meniere’s Disease
This trial compares the effectiveness of methylprednisolone, a corticosteroid medication, with placebo in reducing vertigo attacks in patients with Meniere’s disease. Meniere’s disease is an inner ear disorder causing episodes of spinning dizziness, fluctuating hearing loss, tinnitus, and ear fullness.
Who can participate: Adults over 18 years old with a diagnosis of unilateral Meniere’s disease, meaning the condition affects only one ear, are eligible. Participants must have experienced at least 4 vertigo attacks in the last 6 months. Both males and females can participate.
Who cannot participate: Those without a diagnosis of unilateral Meniere’s disease, outside the specified age range, or part of vulnerable populations unable to give consent are excluded. Other medical conditions that might interfere with the study, current participation in another trial, allergies to the study medication, recent surgeries, or inability to follow study procedures also prevent participation.
What the trial involves: Participants receive injections of either methylprednisolone or placebo directly into the ear through a method called intratympanic injection. The study monitors the frequency of vertigo attacks over one year and assesses quality of life, including aspects related to dizziness and tinnitus. Regular check-ups and questionnaires help track symptom changes and treatment response.
Investigational treatment: Methylprednisolone is a corticosteroid that reduces inflammation and swelling in the inner ear, potentially decreasing the frequency of vertigo episodes by targeting the affected area directly.
Study on Gene Therapy AAVAnc80-hOTOF for Patients with Sensorineural Hearing Loss from Otoferlin Gene Mutations
This Phase 1/2 trial evaluates the safety, tolerability, and effectiveness of different doses of AAVAnc80-hOTOF gene therapy in individuals with sensorineural hearing loss caused by otoferlin gene mutations. The therapy uses an adeno-associated viral vector to deliver a healthy version of the otoferlin gene directly to the inner ear.
Who can participate: Children and adolescents are eligible based on different age groups: one group includes those aged 7-17 years, while other groups include those aged 2-17 years at the time of treatment. Participants must have confirmed mutations in both copies of the OTOF gene through genetic testing and show profound bilateral sensorineural hearing loss on auditory brainstem response testing. Normal otoacoustic emissions must be present in the study ear. Participants of childbearing potential must agree to use effective birth control for one year after treatment. The participant or their guardian must agree to all trial requirements and participate in a follow-up study after trial completion.
Who cannot participate: Those without otoferlin gene-mediated hearing loss, outside the specified age range, or not meeting specific gender requirements if applicable are excluded. Vulnerable populations may be excluded unless specific provisions are made.
What the trial involves: The gene therapy is administered as a single injection directly into the inner ear. Participants are monitored for adverse effects and undergo regular hearing assessments, including auditory brainstem response testing, to evaluate hearing improvements. The trial includes a long-term follow-up protocol after completion to monitor ongoing effects and safety. The study evaluates different dose levels to find the optimal balance between effectiveness and safety.
Investigational treatment: AAVAnc80-hOTOF uses a specially designed virus to deliver a functional copy of the otoferlin gene to inner ear cells, aiming to restore normal hearing function.
Study on SENS-401 to Prevent Hearing Loss Caused by Cisplatin in Adults with Cancer
This trial investigates whether SENS-401 can prevent hearing damage caused by cisplatin, a chemotherapy drug used to treat cancer. Cisplatin can damage the inner ear, leading to hearing loss that often affects high-frequency sounds.
Who can participate: Adults aged 18 or older with cancer who are scheduled to receive chemotherapy including cisplatin are eligible. The cisplatin dose must be at least 70 mg/m² per cycle with a total dose of at least 210 mg/m². Participants must be able to provide informed consent and meet specific hearing test criteria: hearing threshold of 30 decibels or less at 500 Hz, 40 dB or less at 1-2 kHz, 60 dB or less at 4-6 kHz, and 80 dB or less at 8 kHz. Females must not be pregnant or breastfeeding and must agree to use birth control for at least 30 days after the last study drug dose or 6 months after the last cisplatin dose. Males must also agree to use birth control and not donate sperm for the same periods. The treating doctor must believe the participant has a life expectancy of at least 6 months.
Who cannot participate: Those who have not experienced cisplatin-induced hearing loss, are outside the specified age range, or are part of vulnerable populations requiring special protection are excluded.
What the trial involves: Participants take SENS-401 tablets at a dose of 43.5 mg twice daily for up to 23 weeks while receiving cisplatin chemotherapy. Some participants receive placebo instead to allow comparison. Hearing is assessed using audiograms measuring hearing thresholds at frequencies between 0.5 and 12.5 kHz, with the main assessment occurring 4 weeks after the last cisplatin treatment. The study monitors whether SENS-401 effectively protects against hearing loss by comparing hearing changes from baseline.
Investigational treatment: SENS-401 is an otoprotective agent taken orally that works by protecting inner ear cells from damage caused by toxic substances like cisplatin.
Summary
These 5 ongoing clinical trials represent diverse approaches to addressing different types of hearing loss. Three trials focus on gene therapy for otoferlin gene mutations, with two using AAVAnc80-hOTOF and one using DB-OTO. Spain hosts the most trials with three studies, while Germany, France, and the Netherlands each host one. The concentration of gene therapy trials for otoferlin-related hearing loss highlights the growing focus on genetic approaches to treating inherited forms of deafness. Additionally, one trial addresses prevention of chemotherapy-induced hearing loss, and another examines treatment for Meniere’s disease, demonstrating the variety of causes and potential interventions being explored. The age ranges vary considerably, from infants to adults, reflecting the different populations affected by various types of hearing conditions.
