CLTW888A12401

JRCT ID: jRCT2033230516

Registered date:18/12/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: biallelic RPE65 mutation-associated retinal dystrophy
• Date of first enrollment: 30/08/2023
• Target sample size: 15
• Countries of recruitment: Argentina,Japan,Australia,Japan,Austria,Japan,Belgium,Japan,Brazil,Japan,Bulgari,Japan,Canada,Japan,Croatia,Japan,Czechia,Japan,Denmark,Japan,Estonia,Japan,France,Japan,Germany,Japan,Greece,Japan,Hungary,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Korea,Japan,Democratic People's Republic of,Japan,Latvia,Japan,Lithuania,Japan,Netherlands,Japan,Norway,Japan,Poland,Japan,Portugal,Japan,Saudi Arabia,Japan,Slovakia,Japan,Spain,Japan,Sweden,Japan
• Study type: Observational

Outcome(s)

• Primary Outcome: 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 Outcome

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)

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include 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.

• Exclude criteria: None

Related Information

• Primary Sponsor: Sugimoto Toshiya