Clinical trials: Hearing and Vision Loss
Wolfram Syndrome
Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1
Wolfram syndrome (WFS:OMIM 222300) is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy leading to loss of vision, deafness and often diabetes insipidus.
- Trial name
- NCT05659368
- Testing
- Tirzepatide
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Ospedale San Raffaele
Usher Syndrome
Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A
Inherited retinal diseases (IRDs) are a group of degenerative disorders that cause progressive vision loss. Retinitis pigmentosa (RP) is the most common form, with a global prevalence of approximately 1 in 4,500.
- Trial name
- MYO7A (NCT07278843)
- Testing
- Vision tests, Retinal imaging, Questionnaires
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Childhood Cancer
Patients At Risk for Sensory Screening (HPARSS) to Enhance Sensory Deficit Screening in Childhood Cancer Survivors
The overall goal of this study is to attempt to overcome the organizational barriers that impede prompt screening for at-risk sensory deficits in childhood cancer survivors (CCS).
- Trial name
- NCT05582551
- Testing
- Highlighting Patients at Risk for Sensory Screening (HPARSS)
- Where
- United States
- Volunteers
- 146 people
- Sponsor
- Washington University School of Medicine
Hearing Loss, Low Vision
Self-management for Dual Sensory Impaired
This study aims to determine the effectiveness of a self-management program for elderly with an acquired and concurrent hearing and visual impairment due to age-related disease processes.
- Trial name
- NCT01217502
- Testing
- Self management program
- Where
- 4 sites in Netherlands
- Volunteers
- 89 people
- Sponsor
- Radboud University Medical Center
Retinitis Pigmentosa, Usher Syndrome Type 2
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
This clinical trial is a single-site, 30 patient study for participants who have early stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). Funding Source - FDA OOPD and Foundation Fighting Blindness.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
- Trial name
- NCT01530659
- Testing
- NT-501
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Neurotech Pharmaceuticals
Delirium
Improve the Patients' Recovery With Family- Caregivers to End Delirium
This research is being done to find out if patients and caregivers who use the iPREPARED mobile health technology experience less delirium, a type of acute confusion, and if they do experience delirium, the delirium will be less…
- Trial name
- iPREPARED (NCT05118867)
- Testing
- iPREPARED
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Neck Pain, Unilateral Blindness
Unilateral Blindness/ Unilateral Deafness-relation to Neck Pain
Neck pain is a common complaint in the general population affecting 10-15% of the general population. females more than men. Known risk factors are: Trauma, Repetitive lifting of heavy objects at work, driving vibrating vehicles…
- Conditions
- Neck Pain, Unilateral Blindness, Unilateral Deafness
- Trial name
- NCT00589407
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- Hadassah Medical Organization
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
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)
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic…
- Trial name
- NCT06792500
- Testing
- Glycerol Tributyrate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- George Washington University
Usher Syndrome
Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe
This study aims to characterize Usher patients in order to correlate this data with genetic information. Tasks: Standardization and improvement of Usher syndrome diagnosis: refine and elaborate special tests of visual and…
- Trial name
- EURUSH (NCT01954953)
- Where
- 6 sites in France, Germany and 2 more
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
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
Retinitis Pigmentosa, Usher Syndrome Type 2
An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with…
- Trial name
- HELIA (NCT05085964)
- Testing
- RNA antisense oligonucleotide for intravitreal injection
- Where
- 7 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Laboratoires Thea
Angiokeratomas, Chronic Kidney Disease
Biomarker for Patients With Fabry Disease (BioFabry)
Development of a new mass spectrography-based biomarker for the early and sensitive diagnosis of Fabry disease from the blood
- Conditions
- Angiokeratomas, Chronic Kidney Disease, Ocular Abnormalities, Hearing Loss
- Trial name
- BioFabry (NCT02778295)
- Where
- 3 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
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
Usher Syndromes
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.
- Trial name
- SLO RP (NCT04355689)
- Testing
- NPI-001
- Where
- 4 sites in Australia
- Volunteers
- 49 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
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
Usher Syndrome, Congenital Deafness
Clinical and Genetic Testing of Patients With Usher Syndrome
This study is aimed to characterize Russian population of Usher patients.
- Conditions
- Usher Syndrome, Congenital Deafness, Retinitis Pigmentosa
- Trial name
- NCT03319524
- Where
- 6 sites in Russia
- Volunteers
- 28 people
- Sponsor
- Sensor Technology for Deafblind
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Cognitive Impairment, Mood Disorders
Performances of a Brief Assessment Tool for the Early Diagnosis of Geriatric Syndromes by Primary Care Physicians
The AGE (Active Geriatric Evaluation) aims to develop a brief assessment tool adapted to the primary care setting.
- Conditions
- Cognitive Impairment, Mood Disorders, Urinary Incontinence, Malnutrition
- Trial name
- NCT01816087
- Testing
- brief geriatric assessment
- Where
- Switzerland
- Volunteers
- 86 people, healthy volunteers welcome
- Sponsor
- University of Lausanne
Wolfram Syndrome, Fecal Incontinence
Mechanisms of Fecal Incontinence in Wolfram Syndrome
In order to clarify the mechanism of fecal incontinence in in Wolfram syndrome and to characterize its risk factors, the examiner will reviewed the files of patients followed in a Reference Center for Rare Diseases in…
- Trial name
- NCT07313085
- Testing
- Retrospective case-control analysis
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hôpital Necker-Enfants Malades
Retinitis Pigmentosa, Usher Syndrome Type 2
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)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea