Baseline characteristics of adolescent Stargardt disease subjects participating in a Phase 3 study of tinlarebant (DRAGON Trial)
Baseline characteristics of adolescent Stargardt disease subjects participating in a Phase 3 study of tinlarebant (DRAGON Trial)
Early accumulation of cytotoxic byproducts of vitamin A (bisretinoids) is a prominent pathological feature of Stargardt disease (STGD1). Tinlarebant is an orally administered drug that targets plasma retinol binding protein 4 (RPB4) to reduce vitamin A delivery to the eye as a means to reduce bisretinoid accumulation and slow lesion growth in STGD1. A pivotal, Phase 3 study of tinlarebant in adolescent STGD1 patients is ongoing at 20 sites in the USA, Australia, Europe, UK, and Asia (DRAGON, NCT05244304).
Sui, Ruifang
32b79c7d-11d8-4c16-8f84-e8b7fceb2acf
Audo, Isabelle
6941f4f3-9139-4d9c-ba6c-371fe7eec722
Berlin, P.
4d16eb73-f52c-486f-bda7-3275b4e4bc50
Self, James E.
0f6efc58-ae24-4667-b8d6-6fafa849e389
18 June 2025
Sui, Ruifang
32b79c7d-11d8-4c16-8f84-e8b7fceb2acf
Audo, Isabelle
6941f4f3-9139-4d9c-ba6c-371fe7eec722
Berlin, P.
4d16eb73-f52c-486f-bda7-3275b4e4bc50
Self, James E.
0f6efc58-ae24-4667-b8d6-6fafa849e389
et al.
(2025)
Baseline characteristics of adolescent Stargardt disease subjects participating in a Phase 3 study of tinlarebant (DRAGON Trial).
IOVS Investigating Ophthalmology & Visual Science, 66 (8), [1463].
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Meeting abstract
Abstract
Early accumulation of cytotoxic byproducts of vitamin A (bisretinoids) is a prominent pathological feature of Stargardt disease (STGD1). Tinlarebant is an orally administered drug that targets plasma retinol binding protein 4 (RPB4) to reduce vitamin A delivery to the eye as a means to reduce bisretinoid accumulation and slow lesion growth in STGD1. A pivotal, Phase 3 study of tinlarebant in adolescent STGD1 patients is ongoing at 20 sites in the USA, Australia, Europe, UK, and Asia (DRAGON, NCT05244304).
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Published date: 18 June 2025
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Local EPrints ID: 510631
URI: http://eprints.soton.ac.uk/id/eprint/510631
PURE UUID: 96a42af3-b6f7-4f7d-98e8-bc1d8746821c
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Date deposited: 14 Apr 2026 16:46
Last modified: 15 Apr 2026 01:41
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Author:
Ruifang Sui
Author:
Isabelle Audo
Author:
P. Berlin
Corporate Author: et al.
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