Clinical trials: Neonatal Hearing Impairment
Ototoxicity
SPI-1005 for Prevention and Treatment of Tobramycin Induced Ototoxicity
The primary objective of this study is to determine the safety and efficacy of SPI-1005 treatment in CF patients with active pulmonary exacerbation that are receiving an IV course of tobramycin, determined by comparing hearing…
- Trial name
- NCT02819856
- Testing
- SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sound Pharmaceuticals, Incorporated
Cancer, Cardiovascular Disease
NCGENES: North Carolina Clinical Genomic Evaluation by NextGen Exome Sequencing
This study is part of a larger consortium project investigating the validity and best use of next-generation sequencing (in particular, whole exome sequencing, or WES) in clinical care.
- Conditions
- Cancer, Cardiovascular Disease, Neurologic Dysfunction, Congenital Abnormalities
- Trial name
- NCGENES (NCT01969370)
- Testing
- Experimental
- Where
- United States
- Volunteers
- 645 people
- Sponsor
- University of North Carolina, Chapel Hill
Retinal Disease, Age-Related Macular Degeneration
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
- Conditions
- Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
- Trial name
- SCOTS2 (NCT03011541)
- Testing
- Arm 1
- Where
- 4 sites in United States, Austria and 1 more
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Deafness, Kidney Diseases
Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors
The von Hippel Lindau (VHL) gene has recently been identified as the genetic defect resulting in a syndrome of multiple neoplasias.
- Conditions
- Deafness, Kidney Diseases, Kidney Neoplasms, Neoplasms
- Trial name
- NCT00001668
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Retinitis Pigmentosa
Oral N-acetylcysteine for Retinitis Pigmentosa
Retinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several mistakes in the genetic code. Such mistakes are called mutations.
- Trial name
- NAC Attack (NCT05537220)
- Testing
- N-acetylcysteine
- Where
- 31 sites in United States, Austria and 5 more
- Volunteers
- 485 people
- Sponsor
- Johns Hopkins University
Fragile X Syndrome
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age
Individuals with Fragile X Syndrome show differences in how they understand and learn language from infancy. They frequently have lifelong delays in speech and language as well.
- Trial name
- NCT05957549
- Testing
- Speech discrimination
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
CHARGE Syndrome
Neurovisual Function in CHARGE Syndrome
The rationale of the study is to analyze visual function and correlate them with adaptive functions in patients with CHARGE syndrome in order to identify a pattern of specific indicators to enable better patient care
- Trial name
- NCT05764980
- Testing
- visual function evaluation
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Retinitis Pigmentosa (RP), Usher Syndrome
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- SLORP Preserve (NCT07710196)
- Testing
- NPI-001
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Retinitis Pigmentosa, Cone Dystrophy
Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.
Patients with retina dystrophies (retinitis pigmentosa, cone>rods dystrophies, Usher and syndromic) will be correlated with genotype and validate inheritance mode by segregation analysis.
- Conditions
- Retinitis Pigmentosa, Cone Dystrophy, Usher Syndromes, Retina; Dystrophy
- Trial name
- NCT03990727
- Testing
- Retina Analysis-mosaic, Autofluorescence, OCT- 1 micra
- Where
- Mexico
- Volunteers
- 17,000 people, healthy volunteers welcome
- Sponsor
- MejoraVisionMD
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
Mitochondrial Disease
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life.
- Trial name
- NCT05241262
- Testing
- N-Acetylcysteine
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Michio Hirano, MD
Wolfram Syndrome
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
Open label, phase II study non randomized single group assignment of 20 evaluable patients 13 years and older, over 37,5 kg body-weight, with sensorineural hearing loss of at least 20 dB at 8 kHz in high frequency average (HFA)…
- Trial name
- AUDIOWOLF (NCT04940572)
- Testing
- Depakine
- Where
- 2 sites in France, Spain
- Volunteers
- 23 people
- Sponsor
- Centre d'Etude des Cellules Souches
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
Waardenburg Syndrome, Large Vestibular Aqueduct Syndrome
Development and Clinical Application of Two New Genetic Deafness Gene Diagnostic Kit
The purpose of this study is to develop and applicate two new genetic deafness gene diagnostic kit for Waardenburg syndrome and large vestibular aquduct syndrome.
- Trial name
- NCT02418936
- Testing
- gene diagnostic kit
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Xiangya Hospital of Central South University
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Wolfram Syndrome
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome.
- Trial name
- NCT03717909
- Testing
- Sodium Valproate 200Mg E/C Tablet
- Where
- 6 sites in France, Poland and 2 more
- Volunteers
- 63 people
- Sponsor
- University of Birmingham
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)
Hearing Loss, Gentamicin Adverse Reaction
Auditive and Renal Long Term Outcomes - Risk After Aminoglycoside Therapy in Neonates (AURORA)
Gentamicin, in combination with a beta-lactam antibiotic, is commonly used for treatment of neonatal sepsis. Neonates have a high volume of distribution.
- Conditions
- Hearing Loss, Gentamicin Adverse Reaction, Renal Tubular Disorder
- Trial name
- AURORA (NCT03253614)
- Testing
- Audiometry, Urine biomarkers for renal tubular function, Blood pressure
- Where
- Norway
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- University Hospital of North Norway
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients
The Nucleus 24 Auditory Brainstem Implant (ABI) is the only FDA approved device for restoration of meaningful hearing in Neurofibromatosis Type 2 (NF2) patients.
- Trial name
- NCT02589912
- Testing
- Nucleus Profile ABI541 Auditory Brainstem Implant
- Where
- United States
- Sponsor
- Massachusetts Eye and Ear Infirmary
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of…
- 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
- Trial name
- KH176-301 (NCT06451757)
- Testing
- Sonlicromanol
- Where
- 10 sites in United States, Denmark and 5 more
- Volunteers
- 220 people
- Sponsor
- Khondrion BV