Prospective study on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna®) in patients with RPE65-associated inherited retinal degenerations

Phase 1

• Conditions: RPE65-associated inherited retinal degeneration; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2021-000265-33-NL
• Lead Sponsor: The Rotterdam Eye Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-29
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

- Informed consent.
- Clinical diagnosis of inherited retinal disease 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 full-field stimulus test.
- Scheduled to receive treatment with Luxturna®.

Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- None specified.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified