Prospective study on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna®) in patients with RPE65-associated inherited retinal degenerations
- Conditions
- RPE65-associated inherited retinal degenerations (IRDs).
- Registration Number
- NL-OMON22976
- Lead Sponsor
- The Rotterdam Eye Hospital
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 20
Inclusion Criteria
Be able to cooperate.
- Informed consent.
- Clinical diagnosis of IRD with confirmed bi-allelic RPE65 mutations.
- Clinical evidence of viable retinal tissue (RPE cells, photoreceptors, and downstream ganglion cells) as target. Patients have to have more than one
characteristic:
1. total retinal thickness > 100 µm in the posterior pole (OCT).
2. area without atrophy of at least three disc diameters (funduscopy).
3. residual island in the central visual field (within 30º of central fixation; Goldmann).
- Recordable FST.
- Scheduled to receive treatment with Luxturna®.
Exclusion Criteria
- None specified.
Study & Design
- Study Type
- Observational non invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method