Safety Registry for Patients Treated with Luxturna
Research type
Research Study
Full title
A Post-Authorization, Multicenter, Multinational, Longitudinal,Observational Safety Registry Study for Patients Treated with Voretigene Neparvovec (Luxturna)
IRAS ID
278739
Contact name
Robert Henderson
Contact email
Sponsor organisation
Novartis Pharma AG.
Duration of Study in the UK
8 years, 8 months, 21 days
Research summary
The objective of this post-authorization observational study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec. Voretigene neparvovec is a gene therapy developed to restore functional vision in adult and paediatric patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It was approved in the United States and European Union (EU) under the brand name Luxturna®.
Safety will be assessed through collection of all adverse events (AEs) and serious adverse events(SAEs) as well as adverse events of special interest (AESIs) queried through a standardized safety questionnaire. In addition visual function will be assessed over time by means of visual acuity (VA), visual field (VF), full-field light sensitivity threshold testing (FST), optical coherence tomography (OCT), and by patient-reported outcome questionnaires.
Every effort will be made to enroll all patients treated with voretigene neparvovec, and it is expected that a minimum of 40 adult or paediatric, male or female patient can be enrolled globally over a five year enrollment period. Patients will be followed until five years following administration of voretigene neparvovec.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
20/WM/0176
Date of REC Opinion
13 Jul 2020
REC opinion
Further Information Favourable Opinion