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Showing 1–24 of 61 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Congenital Deafness Clear all
Completed Phase 1Phase 2 Interventional Results available

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

NCT02829268
View directory record Official record
Conditions
Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
Interventions
dantrolene sodium
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
5 Years to 60 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Terminated Phase 2 Interventional Results available

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

NCT05085964
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2
Interventions
RNA antisense oligonucleotide for intravitreal injection
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
4
States / cities
Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Determine the Incidence of Long QT Amongst a Large Cohort of Subjects Diagnosed With Unilateral or Bilateral Sensorineural Hearing Loss.

NCT02082431
View directory record Official record
Conditions
Sensorineural Hearing Loss, Long QT Syndrome
Interventions
Not listed
Lead sponsor
Pediatrix
Other
Eligibility
1 Day to 90 Days
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
12
States / cities
Anchorage, Alaska • Phoenix, Arizona • Waterford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2020 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Molecular Genetics of Retinal Degenerations

NCT00231010
View directory record Official record
Conditions
Retinal Degeneration, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
4 Years and older
Enrollment
3,549 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2016
U.S. locations
3
States / cities
Bethesda, Maryland • Boston, Massachusetts • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Not applicable Interventional Results available

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779
View directory record Official record
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Not applicable Interventional

Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis

NCT07420751
View directory record Official record
Conditions
NF2-related Schwannomatosis
Interventions
Auto-captioning Glasses
Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational

Genetic Studies of Tone Deafness

NCT00006076
View directory record Official record
Conditions
Healthy, Tone Deafness
Interventions
Not listed
Lead sponsor
National Institute on Deafness and Other Communication Disorders (NIDCD)
NIH
Eligibility
15 Years and older
Enrollment
968 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Not applicable Interventional

Validating a Self-fitting Hearing Aid

NCT05246904
View directory record Official record
Conditions
Hearing and Vision Loss
Interventions
Fitting algorithm
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2023 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational

Clinical Study of Muenke Syndrome (FGFR3-Related Craniosynostosis)

NCT00106977
View directory record Official record
Conditions
Craniosynostosis, Muenke Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2020
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 25, 2020 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 1Phase 2 Interventional

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

NCT03780257
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
QR-421a, Sham-procedure (dose cohort 1&2 only)
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
4
States / cities
Boston, Massachusetts • Ann Arbor, Michigan • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2022 · Synced Sep 16, 2026, 10:44 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

NCT05158296
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
Ultevursen, Sham-procedure
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
10
States / cities
San Diego, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Not listed Phase 2 Interventional

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

NCT01773278
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions
Antioxidants, Cholesterol
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Up to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting No phase listed Observational

Adaptive Optics Imaging of Outer Retinal Diseases

NCT05355415
View directory record Official record
Conditions
Retinal Degeneration, Age-Related Macular Degeneration, Retinitis Pigmentosa, Hydroxychloroquine Retinopathy, Usher Syndromes, Late-Onset Retinal Degeneration, Cone Dystrophy, Cone Rod Dystrophy, Rod Cone Dystrophy, Rod Dystrophy
Interventions
Adaptive optics imaging
Device
Lead sponsor
Food and Drug Administration (FDA)
Federal
Eligibility
21 Years and older
Enrollment
100 participants
Timeline
2021 – 2028
U.S. locations
2
States / cities
Bethesda, Maryland • Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

NCT05788536
View directory record Official record
Conditions
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Interventions
DB-OTO
Genetic
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
9
States / cities
Los Angeles, California • San Diego, California • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Promoting Early Diagnosis of Congenital Hearing Loss With Patient Navigators

NCT01917747
View directory record Official record
Conditions
Congenital Hearing Loss
Interventions
Patient Navigator, Standard scheduling and follow-up
Behavioral
Lead sponsor
Matthew Bush, MD
Other
Eligibility
Not listed
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
2
States / cities
Lexington, Kentucky • Louisville, Kentucky
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Sep 16, 2026, 10:44 AM EDT
Completed Phase 1 Interventional Results available

A Safety Study of the Auditory Brainstem Implant for Pediatric Profoundly Deaf Patients

NCT02102256
View directory record Official record
Conditions
Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
Interventions
Auditory Brainstem Implant
Device
Lead sponsor
Laurie Eisenberg
Other
Eligibility
2 Years to 6 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 14, 2025 · Synced Sep 16, 2026, 10:44 AM EDT
Completed No phase listed Observational

I-Tracking Neurodegeneration in Early Wolfram Syndrome

NCT03951298
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day and older
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 5, 2024 · Synced Sep 16, 2026, 10:44 AM EDT
Terminated No phase listed Observational

Degenerative Consequences of Congenital Deafness

NCT03866850
View directory record Official record
Conditions
Congenital Deafness
Interventions
Phase duration
Behavioral
Lead sponsor
East Carolina University
Other
Eligibility
15 Years and older
Enrollment
34 participants
Timeline
2019 – 2023
U.S. locations
1
States / cities
Greenville, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 6, 2023 · Synced Sep 16, 2026, 10:44 AM EDT
Active, not recruiting Phase 2 Interventional

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

NCT06627179
View directory record Official record
Conditions
Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
10
States / cities
San Francisco, California • Miami, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting Phase 1 Interventional

Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

NCT05241262
View directory record Official record
Conditions
Mitochondrial Disease
Interventions
N-Acetylcysteine
Drug
Lead sponsor
Michio Hirano, MD
Other
Eligibility
18 Years to 80 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Sep 16, 2026, 10:44 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients

NCT02589912
View directory record Official record
Conditions
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Interventions
Nucleus Profile ABI541 Auditory Brainstem Implant
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years to 80 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 5, 2019 · Synced Sep 16, 2026, 10:44 AM EDT
Recruiting Not applicable Interventional

Stem Cell Ophthalmology Treatment Study II

NCT03011541
View directory record Official record
Conditions
Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions
Arm 1
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Sep 16, 2026, 10:44 AM EDT