Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
Who is this study for? Patients with retinitis pigmentosa due to mutations in PDE6B gene
What treatments are being studied? AAV2/5-hPDE6B
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B. At least twelve patients 18 years of age or older, within four consecutive cohorts of patients, will be recruited. Then at least four patients 13 years of age or older, within a fifth cohort, will be recruited.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 13
Healthy Volunteers: f
View:
• Clinical and molecular diagnosis of retinitis pigmentosa caused by defect in PDE6B gene without other syndromic manifestations
• Aged above 13 years
• Ability to give informed consent
Locations
Other Locations
France
Clinique Ophtalmologique, CHU de Nantes
RECRUITING
Nantes
Contact Information
Primary
Medical Director / Chief Development Officer, MD
contact@eyednatx.com
01 81 69 87 70
Time Frame
Start Date: 2017-11-06
Estimated Completion Date: 2029-12
Participants
Target number of participants: 23
Treatments
Experimental: Cohort 1 - Low Dose
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the lowest dose. Dose-escalation will be performed after DSMC assessment.
Experimental: Cohort 2a - Medium Dose
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the medium dose. Confirmatory dose will be determined after DSMC assessment.
Experimental: Cohort 2b - High Dose
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the highest dose. Confirmatory dose will be determined after DSMC assessment.
Experimental: Cohort 3 - High Dose (confirmatory cohort)
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.
Experimental: Cohort 4 - High Dose - 13 years old or older population
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.
Related Therapeutic Areas
Sponsors
Leads: eyeDNA Therapeutics