Usher SyndromeNCT04906135
Auditory Neural Function in Implanted Patients With Usher Syndrome
A study testing Experimental manipulation of stimulation parameters in people with Usher Syndrome and Cochlear Implantation, run by Ohio State University at 4 sites in United States.
Trial summary
Usher syndrome (USH) causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. As the first step toward developing evidence-based practice for managing implant patients with USH, this study evaluates local neural health, as well as the neural encoding of… Read more about this study
- Who is it for?
- Ages 1 year to 85 years
- Conditions the study is for
- Usher Syndrome, Cochlear Implantation
- What is being tested?
- Experimental manipulation of stimulation parameters
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Oct 2021to 11 Jul 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 85 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Minimum of 6 months of listening experience with cochlear implant
- Diagnosed with Usher syndrome or idiopathic hearing loss
Who cannot join
- Severe medical comorbidities
- Electrode malposition or migration as determined based on imaging results
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Boston children's Hospital
Boston, Massachusetts, 02453
Gina Hounam
Columbus, Ohio, 43205-2664
The Ohio State University
Columbus, Ohio, 43210
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-8718
About this study
Usher syndrome (USH) causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. As the first step toward developing evidence-based practice for managing implant patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations.
Read the detailed description
Usher syndrome (USH) is an autosomal recessive disorder characterized by hearing loss, visual impairment, and in some cases, vestibular dysfunction. It is the leading cause of hereditary deaf-blindness in humans. USH causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. Whereas there is no treatment for arresting this degenerative process or for restoring visual loss, the restoration of auditory input is possible with cochlear implantation. Due to the progressive deterioration in vision, using visual cues for communication will eventually become impossible. Therefore, the importance of optimizing auditory inputs through cochlear implants (CIs) for patients with USH is paramount. However, patients with USH have much higher rates of neurological, mental, or behavioral disorders than the general CI patient population, which limits their ability to provide reliable behavioral responses or sufficient verbal descriptions of their auditory perception, especially for pediatric patients. In addition, optimal programming parameters for CI users with CN damage differ from those used in typical CI users due to declined CN responsiveness to electrical stimulation. As a result, the clinical programming process in implanted patients with USH can be extremely challenging. To date, auditory neural encoding of electrical stimulation in patients with USH has not been systematically evaluated. Consequently, the field lacks evidence-based practice guidelines for managing implanted patients with USH. For patients who cannot provide reliable feedback, clinicians rely on a "trial-and-error" approach for adjusting CI programming settings, which ultimately may not result in appropriate programming maps for individual patients. Therefore, there is an urgent need to develop objective clinical tools for optimizing CI settings for these patients. As the first step toward developing evidence-based practice for managing patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations. Results of this study have high scientific significance because they will establish how CN degeneration affects neural encoding and processing of electrical stimulation, and identify tests that distinguish the loss of spiral ganglion neurons from the loss of peripheral axons. Results of this study also have high clinical significance because they will 1) lay the groundwork for developing effective, evidence-based clinical practice guidelines for managing patients with USH, and 2) yield an objective tool for assessing the site-specific electrode-neuron interface in all CI users, which is foundational for creating optimal programming maps for individual patients.
- Conditions
- Usher Syndrome, Cochlear Implantation
- Being tested
- Experimental manipulation of stimulation parameters
- Keywords
- Auditory Electrophysiology
- Medical subject headings
- Usher Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Experimental manipulation of stimulation parametersOther
This translational mechanistic study involves changing stimulation parameters (i.e., experimental manipulation) for measuring the electrically evoked compound action potential in order to understand the pathophysiology of the auditory system in patients with Usher syndrome.
| Study group | Role | What they receive |
|---|---|---|
| Usher Syndrome | Experimental | Adult and pediatric cochlear implant users with Usher syndrome Receives: Other: Experimental manipulation of stimulation parameters |
| Idiopathic Hearing Loss | Active comparator | Adult and pediatric cochlear implant users with idiopathic hearing loss Receives: Other: Experimental manipulation of stimulation parameters |
What is being measured
The primary outcome is the main question the study is designed to answer.
The electrically evoked compound action potential
The primary outcome measure is the neural response generated by the electrically-stimulated cochlear nerve, which can be considered a health-related, biomedical outcome.
Measured: Outcome measures will be obtained from Day 1 through study completion, an average of 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2021
- Study start1 Oct 2021
- Primary completion31 Mar 2025
- Study completion11 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Usher SyndromeStudies of the same condition, open and under way.
- More trials run by Ohio State UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ohio State University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Responsible party
- Principal investigator, Shuman He, Ohio State University
- Investigators
- Shuman He, MD, PhD, Principal investigator, Ohio State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04906135
- Sponsor's protocol ID
- 2018H0344
- Other IDs
- R21DC019458
- Status last verified
- Jul 2025
- First submitted
- 24 May 2021
- Last updated
- 29 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Auditory Neural Function in Implanted Patients With Usher Syndrome
- ClinicalTrials.gov ID
- NCT04906135
- Acronym / sponsor ID
- 2018H0344
- Conditions
- Usher Syndrome, Cochlear Implantation
- Keywords
- Auditory Electrophysiology
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Ohio State UniversityUniversity, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Study start
- 1 Oct 2021 (actual)
- Primary completion
- 31 Mar 2025 (actual)
- Study completion
- 11 Jul 2025 (actual)
- First posted
- 28 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Usher syndrome (USH) causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. As the first step toward developing evidence-based practice for managing implant patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations.
Detailed description
Usher syndrome (USH) is an autosomal recessive disorder characterized by hearing loss, visual impairment, and in some cases, vestibular dysfunction. It is the leading cause of hereditary deaf-blindness in humans. USH causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. Whereas there is no treatment for arresting this degenerative process or for restoring visual loss, the restoration of auditory input is possible with cochlear implantation. Due to the progressive deterioration in vision, using visual cues for communication will eventually become impossible. Therefore, the importance of optimizing auditory inputs through cochlear implants (CIs) for patients with USH is paramount. However, patients with USH have much higher rates of neurological, mental, or behavioral disorders than the general CI patient population, which limits their ability to provide reliable behavioral responses or sufficient verbal descriptions of their auditory perception, especially for pediatric patients. In addition, optimal programming parameters for CI users with CN damage differ from those used in typical CI users due to declined CN responsiveness to electrical stimulation. As a result, the clinical programming process in implanted patients with USH can be extremely challenging. To date, auditory neural encoding of electrical stimulation in patients with USH has not been systematically evaluated. Consequently, the field lacks evidence-based practice guidelines for managing implanted patients with USH. For patients who cannot provide reliable feedback, clinicians rely on a "trial-and-error" approach for adjusting CI programming settings, which ultimately may not result in appropriate programming maps for individual patients. Therefore, there is an urgent need to develop objective clinical tools for optimizing CI settings for these patients. As the first step toward developing evidence-based practice for managing patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations. Results of this study have high scientific significance because they will establish how CN degeneration affects neural encoding and processing of electrical stimulation, and identify tests that distinguish the loss of spiral ganglion neurons from the loss of peripheral axons. Results of this study also have high clinical significance because they will 1) lay the groundwork for developing effective, evidence-based clinical practice guidelines for managing patients with USH, and 2) yield an objective tool for assessing the site-specific electrode-neuron interface in all CI users, which is foundational for creating optimal programming maps for individual patients.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Usher Syndrome | Experimental | Other: Experimental manipulation of stimulation parametersAdult and pediatric cochlear implant users with Usher syndrome |
| Idiopathic Hearing Loss | Active comparator | Other: Experimental manipulation of stimulation parametersAdult and pediatric cochlear implant users with idiopathic hearing loss |
Who may qualify
- Ages
- 1 year to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Minimum of 6 months of listening experience with cochlear implant
- Diagnosed with Usher syndrome or idiopathic hearing loss
Key exclusion criteria (2 listed)
- Severe medical comorbidities
- Electrode malposition or migration as determined based on imaging results
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Experimental manipulation of stimulation parametersThis translational mechanistic study involves changing stimulation parameters (i.e., experimental manipulation) for measuring the electrically evoked compound action potential in order to understand the pathophysiology of the auditory system in patients with Usher syndrome. | Other | Idiopathic Hearing Loss, Usher Syndrome |
Primary outcome
The electrically evoked compound action potential
Time frame: Outcome measures will be obtained from Day 1 through study completion, an average of 2 years.
The primary outcome measure is the neural response generated by the electrically-stimulated cochlear nerve, which can be considered a health-related, biomedical outcome.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2025.
Related trials for Usher Syndrome
RecruitingPhase 1/2
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
RecruitingPhase 3
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
Retinitis Pigmentosa (RP), Usher Syndrome
Not yet recruitingPhase 2
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Active, not recruitingObservational study
Rate of Progression in USH2A-related Retinal Degeneration
Usher Syndrome, Type 2A, Retinitis Pigmentosa 39
Active, not recruitingPhase 2
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Retinitis Pigmentosa (RP), Usher Syndrome Type 2
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shuman He, MD, PhD principal investigator
Ohio State University
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Experimental manipulation of stimulation parameters in people with Usher Syndrome and Cochlear Implantation, run by Ohio State University at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The electrically evoked compound action potential (time frame: Outcome measures will be obtained from Day 1 through study completion, an average of 2 years.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2025.