To collect long-term, real world data on safety and efficacy of gene therapy with voretigene neparvovec (Luxturna®).
ID
Source
Brief title
Condition
- Congenital eye disorders (excl glaucoma)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Full-field stimulus testing (FST) at 1 year.
Secondary outcome
Visual acuity
Visual field
Mobility course
Background summary
RPE65-associated inherited retinal degenerations (IRDs) are rare, and account
for 5*10% of all autosomal recessive childhood-onset IRDs. Visual function of
these patients can vary early in life, but inevitably deteriorates towards
blindness. Gene therapy with voretigene neparvovec (Luxturna®) was approved by
the US Food and Drug Administration (FDA) in 2017 and by the European Medicines
Agency (EMA) in 2018.
Study objective
To collect long-term, real world data on safety and efficacy of gene therapy
with voretigene neparvovec (Luxturna®).
Study design
Multi-center prospective, observational study; follow-up (FU): 5 years.
Study burden and risks
Assessments in this study are part of the clinical routine. Extra time for each
MLMT will be 4h; risks are negligible.
Schiedamse Vest 180
Rotterdam 3011 BH
NL
Schiedamse Vest 180
Rotterdam 3011 BH
NL
Listed location countries
Age
Inclusion criteria
Scheduled to receive treatment with Luxturna®
Informed consent
Exclusion criteria
None
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2021-000265-33-NL |
| CCMO | NL76234.000.21 |
| OMON | NL-OMON22976 |