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

Status: Recruiting
Location: See location...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Healthy Volunteers: f
View:

• Age: from 5 years of age

• Patient with PDE6A, PDE6B, and RHO-based retinitis pigmentosa

• Patient and/or legal representatives are willing and able to give written informed consent

Locations
Other Locations
Germany
Institute for Ophthalmic Research, University Tübingen
RECRUITING
Tübingen
Contact Information
Primary
Katarina Stingl, Prof
katarina.stingl@med.uni-tuebingen.de
070712988088
Time Frame
Start Date: 2023-11-17
Estimated Completion Date: 2027-03
Participants
Target number of participants: 40
Treatments
PDE6A patients
15 patients with mutation in PDE6A
PDE6B patients
15 patients with mutation in PDE6B
RHO patients
10 patients with mutation in RHO
Sponsors
Leads: University Hospital Tuebingen

This content was sourced from clinicaltrials.gov