CLTW888A12401
Not Applicable
- Conditions
- biallelic RPE65 mutation-associated retinal dystrophy
- Registration Number
- JPRN-jRCT2033230516
- Lead Sponsor
- Sugimoto Toshiya
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 15
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
None
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Measures
Name Time Method The secondary objectives of this safety registry study are:<br> .To follow pregnancy outcomes in patients (and female partners of patient) who received voretigene neparvovec<br> .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)