Natural History Study of Usher Syndrome in a Cohort of Patients Followed Longitudinally for 5 Years

Status: Recruiting
Location: See all (4) locations...
Study Type: Observational
SUMMARY

Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the foreseen USH clinical trials. The clinical arm of the project aims at performing a deep-phenotyping of retinal degeneration, hearing loss, vestibular dysfunction, neurocognitive ability of subects with a molecular diagnosis of any Usher syndrome. Functional and structural parameters for retinal, auditory, and vestibular impairments are followed overtime to document the natural history of the disease and establish relevant clinical endpoint for disease progression that may be useful for future clinical trials.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Patient with a molecular diagnosis of Usher syndrome type I, II or III or a clinical diagnosis of Usher syndrome type I, II or III which will then be confirmed by a molecular diagnosis

• Health insurance beneficiary

• Informed consent signed by the patient or their legal representatives

Locations
Other Locations
France
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
RECRUITING
Paris
CHU Necker
RECRUITING
Paris
CHU Pitié Salpêtrière
RECRUITING
Paris
CHU Robert Debré
RECRUITING
Paris
Contact Information
Primary
Isabelle AUDO, Pr
isabelle.audo@inserm.fr
0140021430
Backup
Thilissa DIB
tdib@15-20.fr
0140021455
Time Frame
Start Date: 2017-06-08
Estimated Completion Date: 2027-06-08
Participants
Target number of participants: 400
Sponsors
Leads: Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Collaborators: Assistance Publique - Hôpitaux de Paris

This content was sourced from clinicaltrials.gov