CLTW888A12401

A Post-Authorization, Multicenter, Multinational, Longitudinal, Observational Safety Registry Study for Patients Treated with Voretigene Neparvovec

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT2033230516

Enrollment

Registered

2023-12-18

Start date

2023-08-30

Completion date

Unknown

Last updated

2026-06-29

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

Conditions

biallelic RPE65 mutation-associated retinal dystrophy

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Is planning to receive or has received voretigene neparvovec in at least one eye. 2. Provided informed consent/assent to participate in this study. 3. Has not previously participated and is not currently participating in an interventional clinical trial with voretigene neparvovec.

Exclusion criteria

Exclusion criteria: None

Design outcomes

Primary

Measure	Time frame
The primary objective for this safety registry study for voretigene neparvovec is to collect all AESIs, as well as any other AEs and SAEs.	—

Secondary

Measure	Time frame
The secondary objectives of this safety registry study are: .To follow pregnancy outcomes in patients (and female partners of patient) who received voretigene neparvovec .To assess visual function over time (e.g., as measured by visual acuity (VA), visual field (VF), full-field light sensitivity threshold testing (FST)), and optical coherence tomography (OCT)	—

Countries

Argentina, Australia, Austria, Belgium, Brazil, Bulgari, Canada, Croatia, Czechia, Democratic People's Republic of, Denmark, Estonia, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Korea, Latvia, Lithuania, Netherlands, Norway, Poland, Portugal, Saudi Arabia, Slovakia, Spain, Sweden

Contacts

Public ContactNovartis Direct

Novartis Pharma. K.K.

sm.pms@novartis.com+81-120-003-293

Outcome results