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

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

Status
Unknown
Phases
Phase 4
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2021-000265-33-NL
Enrollment
20
Registered
2021-01-29
Start date
Unknown
Completion date
Unknown
Last updated
2021-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

RPE65-associated inherited retinal degeneration

Interventions

Trade Name: Luxturna Product Name: voretigene neparvovec Product Code: EU/1/18/1331/001 Pharmaceutical Form: Concentrate and solvent for solution for injection INN or Proposed INN: VORETIGENE NEPARVOV

Sponsors

The Rotterdam Eye Hospital
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

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

Exclusion criteria: - None specified.

Design outcomes

Primary

MeasureTime frame
Main Objective: To collect long-term, real world data on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna®).;Secondary Objective: Not applicable;Primary end point(s): Full-field stimulus test (FST) at 1 year.;Timepoint(s) of evaluation of this end point: FST at baseline, day 30, day 90, day 180 and years 1, 2, 3, 4, 5.—

Secondary

MeasureTime frame
Secondary end point(s): Best corrected visual acuity, intraocular pressure, kinetic perimetry, optical coherence tomography.;Timepoint(s) of evaluation of this end point: Baseline, day 30, day 90, day 180 and years 1, 2, 3, 4, 5.—

Countries

Netherlands

Contacts

Public ContactRotterdam Ophthalmic Institute

The Rotterdam Eye Hospital

r.wubbels@oogziekenhuis.nl31104023430

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026