Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene
Retinitis Pigmentosa
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring Adeno-associated virus, AAV, Retinitis Pigmentosa, PDE6B, Gene Therapy, Gene Transfer, Retinal Dystrophy, Eye Diseases, Vision Disorders, Eye Diseases, Hereditary, Retinal Diseases, Retinal Degeneration
Eligibility Criteria
Key Inclusion Criteria:
- Clinical and molecular diagnosis of retinitis pigmentosa caused by defect in PDE6B gene without other syndromic manifestations
- Aged 18 years or older
- Ability to give informed consent
Key Exclusion Criteria:
- Previous ocular surgery or thermal laser within 6 months before the surgery
- Lens opacities or obscured ocular media upon recruitment such reliable evaluation or grading of the posterior segment cannot be performed
- Known serious allergies to the fluorescein dye used in angiography, to the mydriatic, steroidal and non-steroidal eye drops
- Participation in another clinical trial with an investigational agent
- Enrolled or being enrolled in another gene therapy clinical trial
- Active, extraocular infection requiring the prolonged or chronic use of antimicrobial agents
- Chronic medical conditions, cancer
- Abnormal laboratory values
- On immunosuppressive therapy
Sites / Locations
- Clinique Ophtalmologique, CHU de NantesRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Experimental
Experimental
Experimental
Experimental
Experimental
Cohort 1 - Low Dose
Cohort 2a - Medium Dose
Cohort 2b - High Dose
Cohort 3 - High Dose (confirmatory cohort)
Cohort 4 - High Dose - 13 years old or older population
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the lowest dose. Dose-escalation will be performed after DSMC assessment.
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the medium dose. Confirmatory dose will be determined after DSMC assessment.
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the highest dose. Confirmatory dose will be determined after DSMC assessment.
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.