Distribution of DDAF Lesions in Stargardt Disease Type 1 (STGD1)
Research type
Research Study
Full title
Non-Interventional Study to Determine the Distribution of Definitely Decreased Autofluorescence (DDAF) Lesions in Children and Adults with Macular Atrophy Secondary to Stargardt Disease Type 1 (STGD1)
IRAS ID
329062
Contact name
Michel Michaelides
Contact email
Sponsor organisation
Alnylam Pharmaceuticals
Duration of Study in the UK
0 years, 6 months, 21 days
Research summary
Stargardt disease is a rare inherited retinal (eye) disease, typically presenting within the first two decades of life. Stargardt Disease Type 1 (STGD1) is the most common form of STGD and is estimated to account for more than 95% of cases. The main symptom is progressive central vision loss in both eyes.
At present, there are no approved treatments for STGD. Current management relies primarily on lifestyle modifications and/or inclusion of low vision aids to maximize the use of vision.
This is a global, non-interventional study to learn about Stargardt disease including how it impacts on daily activities for patients. Participants with STGD1 will undergo eye examinations, retinal imaging techniques including fundus autofluorescence(FAF), visual acuity assessments, patient-reported outcomes questionnaires and participant/caregiver surveys to characterise their disease.
Eligible participants will be 6 years of age or older and have a clinical diagnosis of macular atrophy secondary to Stargardt disease Type 1 (STGD1) with documentation of a minimum of 2 known mutations. The study aims to recruit approximately 50 participants globally.
The study will consist of a single visit, which will include screening and all other assessments. Whilst completing the single study visit within one day will be encouraged, the single visit will have a 7-day window, so that participants can return to complete the visit on another day within the window, if needed.The characterisation of this patient population will hopefully inform the development of future clinical studies of investigational treatments for STGD1.
REC name
Yorkshire & The Humber - Bradford Leeds Research Ethics Committee
REC reference
23/YH/0156
Date of REC Opinion
18 Aug 2023
REC opinion
Further Information Favourable Opinion