Clinical trials: Congenital Hearing Loss
DFNB9, Congenital Hearing Loss
A Study of EH002 Gene Therapy for Otoferlin Gene Mutation-mediated Hearing Loss
The study is designed to evaluate the safety, tolerability, and preliminary efficacy of EH002 for the treatment of congenital deafness caused by mutations in the OTOF gene.
- Conditions
- DFNB9, Congenital Hearing Loss, Hearing Loss, Sensorineural
- Trial name
- NCT06722170
- Testing
- EH002 administration
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Yilai Shu
Congenital Deafness
Degenerative Consequences of Congenital Deafness
In this study, the investigators will study one of the basic biophysical properties of the auditory nerve, charge integration, behaviorally (detection threshold versus phase duration functions).
- Trial name
- NCT03866850
- Testing
- Phase duration
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- East Carolina University
Retinitis Pigmentosa (RP), Usher Syndrome Type 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
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13…
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- CAPELLA (NCT07796646)
- Testing
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Volunteers
- 81 people
- Sponsor
- Laboratoires Thea
Hearing Loss, Genetic Disease
Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)
This cohort study aims to explore the trends and differences in multidimensional perceptual levels of patients after cochlear implants or gene therapy, as well as to comprehensively assess the efficacy of gene therapy for…
- Conditions
- Hearing Loss, Genetic Disease, Speech Perception
- Trial name
- NCT06237790
- Where
- 6 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Eye & ENT Hospital of Fudan University
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Retrocochlear Pathology, Auditory Processing Disorder, Central
Cochlear Implantation in Single Sided Deafness and Asymmetrical Hearing Loss: a Cost/Utility Study.
The investigators assume that cochlear implants in this indication are not only effective but also cost-effective. The investigators' experimental protocol relies on real life therapeutic strategy, where a cochlear implant may be…
- Conditions
- Retrocochlear Pathology, Auditory Processing Disorder, Central, Major Cochlear Ossification or Malformation
- Trial name
- CISSD (NCT02204618)
- Testing
- cochlear implantation, 6 months initial abstention
- Where
- France
- Volunteers
- 160 people
- Sponsor
- University Hospital, Toulouse
Wolfram Syndrome
AMX0035 in Adult Patients With Wolfram Syndrome
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
- Trial name
- NCT05676034
- Testing
- AMX0035
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Retinitis Pigmentosa Syndromic, Congenital Deafness
Natural History and Genetic Studies of Usher Syndrome
This study will explore clinical and genetic aspects of Usher syndrome, an inherited disease causing deafness or impaired hearing, visual problems, and, in some cases, unsteadiness or balance problems.
- Conditions
- Retinitis Pigmentosa Syndromic, Congenital Deafness, Usher Syndrome, Retinitis Pigmentosa and Deafness
- Trial name
- NCT00106743
- Where
- 2 sites in United States
- Volunteers
- 249 people
- Sponsor
- National Eye Institute (NEI)
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Congenital Hearing Loss
Communities Helping the Hearing of Infants by Reaching Parents
Hearing loss is the most common sensory congenital disorder and this condition is diagnosable and treatable. Children that are born with hearing loss have to undergo several hearing tests to diagnose the condition and many…
- Trial name
- CHHIRP (NCT03875339)
- Testing
- Navigator Guidance
- Where
- 2 sites in United States
- Volunteers
- 2,699 people, healthy volunteers welcome
- Sponsor
- Matthew Bush, MD
Hearing Disorder
Genetic Analyses of Nonsyndromic and Syndromic Deafness in Pakistan
Objective: One objective of this study is to genetically map and identify mutated genes for human hereditary hearing loss. A second objective is to study the function of these genes in the auditory system using mouse models.
- Trial name
- NCT00341874
- Where
- Pakistan
- Volunteers
- 18,009 people
- Sponsor
- National Institute on Deafness and Other Communication Disorders (NIDCD)
Wolfram Syndrome, Diabetes Mellitus
Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study
In this study, the investigators hypothesize that studying monogenic variants with strong effect associated with severe insulin deficiency of Wolfram syndrome will provide important insights into the more complex type 1 and type…
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Deafness
- Trial name
- NCT02841553
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- Washington University School of Medicine
Congenital Hearing Loss, Gene Therapy
Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss
This study aims to establish a novel multimodal evaluation and precision rehabilitation system for patients with congenital hearing loss who have received hearing aids, cochlear implantation, or emerging gene therapy.
- Conditions
- Congenital Hearing Loss, Gene Therapy, Speech Perception, Rehabilitation
- Trial name
- NCT07024524
- Testing
- Rehabilitation training
- Where
- China
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Eye & ENT Hospital of Fudan University
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP)
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor…
- Conditions
- Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD)
- Trial name
- CLARICO (NCT06789445)
- Testing
- OpCT-001
- Where
- 5 sites in United States
- Volunteers
- 54 people
- Sponsor
- BlueRock Therapeutics
Congenital Hearing Loss, Sensorineural Hearing Loss
Alpha-Tectorin, Interleukin-10 and Thiol-Disulfide Homeostasis in Children With Congenital Hearing Loss
Congenital hearing loss is one of the most common sensory disorders of childhood, and the structural, immunologic and oxidative mechanisms involved in its development are incompletely understood.
- Trial name
- NCT07860580
- Testing
- Blood sample collection
- Where
- Turkey (Türkiye)
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Harran University
Retinitis Pigmentosa, Usher Syndrome Type 2
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)
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05158296
- Testing
- Ultevursen
- Where
- 15 sites in United States, Germany and 2 more
- Volunteers
- 7 people
- Sponsor
- Laboratoires Thea
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth
Natural History Study for Charcot Marie Tooth Disease
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden…
- Conditions
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
- Trial name
- NCT05902351
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Hereditary Neuropathy Foundation
Congenital Cytomegalovirus Infection, Sensorineural Hearing Loss
Congenital Cytomegalovirus: Efficacy of Antiviral Treatment in a Randomized Controlled Trial
The objective of the trial is to investigate whether early treatment with oral valganciclovir of infants with both congenital cytomegalovirus infection and sensorineural hearing loss can prevent progression of hearing loss.
- Trial name
- CONCERT (NCT01655212)
- Testing
- Valganciclovir
- Where
- Netherlands
- Volunteers
- 2 people
- Sponsor
- Dr. Ann C.T.M. Vossen
Osteogenesis Imperfecta
Effects of Bisphosphonates on OI-Related Hearing Loss
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in…
- Trial name
- NCT04152551
- Testing
- Risedronate Oral Tablet
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Hospital for Special Surgery, New York
Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome
GLP Analogs for Diabetes in Wolfram Syndrome Patients
Wolfram syndrome, also referred to as DIDMOAD (diabetes insipidus, diabetes mellitus, optic atrophy and deafness) is a genetic syndrome characterized by beta-cell dysfunction and apoptosis leading to diabetes, neurodegeneration…
- Trial name
- NCT01302327
- Testing
- Exenatide
- Sponsor
- Hadassah Medical Organization