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ClinicalTrials.gov public records Last synced Oct 8, 2026, 6:31 AM EDT

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Showing 25–48 of 63 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Hearing Impairment Clear all
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 8, 2024 · Synced Oct 8, 2026, 6:31 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 7, 2024 · Synced Oct 8, 2026, 6:31 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 23, 2026 · Synced Oct 8, 2026, 6:31 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 29, 2026 · Synced Oct 8, 2026, 6:31 AM EDT
Completed Not applicable Interventional

NCGENES: North Carolina Clinical Genomic Evaluation by NextGen Exome Sequencing

NCT01969370
View directory record Official record
Conditions
Cancer, Cardiovascular Disease, Neurologic Dysfunction, Congenital Abnormalities, Hearing Loss
Interventions
Experimental
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated May 3, 2017 · Synced Oct 8, 2026, 6:31 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT06792500
View directory record Official record
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 8, 2026, 6:31 AM EDT
Terminated Phase 2 Interventional Results available

Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants

NCT03107871
View directory record Official record
Conditions
Cmv Congenital, CMV, Congenital Cmv, SNHL, Sensorineural Hearing Loss
Interventions
Valganciclovir, Simple Syrup
Drug
Lead sponsor
Albert Park
Other
Eligibility
1 Month to 12 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
30
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 25 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 8, 2026, 6:31 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 6, 2019 · Synced Oct 8, 2026, 6:31 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 31, 2020 · Synced Oct 8, 2026, 6:31 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 25, 2023 · Synced Oct 8, 2026, 6:31 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 7, 2023 · Synced Oct 8, 2026, 6:31 AM EDT
Completed Phase 2 Interventional

Effectiveness of Therapy Via Telemedicine Following Cochlear Implants

NCT02497690
View directory record Official record
Conditions
Congenital Sensorineural Deafness
Interventions
Telemedicine, In-person ART
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
6 Months to 6 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 19, 2021 · Synced Oct 8, 2026, 6:31 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 12, 2024 · Synced Oct 8, 2026, 6:31 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome

NCT00004345
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1999
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 8, 2026, 6:31 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 5, 2026 · Synced Oct 8, 2026, 6:31 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Tracking Neurodegeneration in Early Wolfram Syndrome

NCT02455414
View directory record Official record
Conditions
Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day and older
Enrollment
101 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 8, 2026, 6:31 AM EDT
Completed Phase 2 Interventional Results available

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

NCT01530659
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
Interventions
NT-501, Sham
Drug · Procedure
Lead sponsor
Neurotech Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 14, 2023 · Synced Oct 8, 2026, 6:31 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 26, 2020 · Synced Oct 8, 2026, 6:31 AM EDT
Completed Phase 1 Interventional

An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors

NCT00030043
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
Penetrating auditory brainstem implant
Device
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
3
States / cities
Los Angeles, California • Pasadena, California • Englewood, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 8, 2026, 6:31 AM EDT
Completed No phase listed Observational Results available

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
128 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 Sep 23, 2026 · Synced Oct 8, 2026, 6:31 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

NCT05176717
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
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
2
States / cities
Dallas, Texas • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 7, 2024 · Synced Oct 8, 2026, 6:31 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

NCT01505062
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa
Interventions
SAR421869
Drug
Lead sponsor
Sanofi
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 28, 2022 · Synced Oct 8, 2026, 6:31 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 15, 2025 · Synced Oct 8, 2026, 6:31 AM EDT
Recruiting Phase 3 Interventional

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

NCT06451757
View directory record Official record
Conditions
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions
Sonlicromanol, Placebo
Drug
Lead sponsor
Khondrion BV
Industry
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 8, 2026, 6:31 AM EDT