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Natural History Study in Patients with PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa
Sponsor: University Hospital Tuebingen
Summary
The aim of the study is to apply a novel clinical investigation protocol in patients with Phosphodiesterase 6A (PDE6A), PDE6B and Rhodopsin (RHO)-based retinitis pigmentosa. This novel, multimodal clinical examination protocol describes and correlates structural, functional and metabolic aspects during natural disease development. Test-retest variability of new measurements as well as correlations of the structural, functional, and metabolic changes will be defined to be able to define well-suited readouts for safety and efficacy of future treatment developments before they reach the clinical phase.
Official title: Exploration of New Sensitive Clinical Readouts and Biomarkers That Can Be Used As Clinical Endpoints Tailored to Monitor Treatment Effects in PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa: a Non-interventional Trial
Key Details
Gender
All
Age Range
5 Years - Any
Study Type
OBSERVATIONAL
Enrollment
40
Start Date
2023-11-17
Completion Date
2027-03
Last Updated
2025-03-30
Healthy Volunteers
No
Conditions
Locations (1)
Institute for Ophthalmic Research, University Tübingen
Tübingen, Baden-Wurttemberg, Germany