POLARIS
Research type
Research Study
Full title
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (POLARIS)
IRAS ID
339659
Contact name
Mariya Moosajee
Contact email
Sponsor organisation
Splice Bio SL
Duration of Study in the UK
3 years, 9 months, 23 days
Research summary
Stargardt Disease Type 1 (STGD1) is a rare, progressive disease which leads to visual impairment and central vision loss. STGD1 generally occurs in childhood or adolescence but can occur into adulthood. Currently there are no approved treatments or cure for STGD1.
This is an observational study, funded by Splice Bio, monitoring the progression of STGD1. The purpose of this study is to observe and gather information from patients with STGD1 to identify the common signs and symptoms of the disease which will then aid in the future development of treatments.
Approximately 75 participants, aged 12 to 65, will be enrolled in this study across 20 centres globally. The study will consist of a screening phase (to ensure participants meet all eligibility criteria to take part in the study) and an observational phase and will run for up to 96 weeks for each participant.
The primary study objective is to evaluate disease progression through various eye examinations and questionnaires.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
24/EM/0075
Date of REC Opinion
29 Apr 2024
REC opinion
Further Information Favourable Opinion