LBS-008-CT07
Research type
Research Study
Full title
A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects with Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects with Stargardt Disease
IRAS ID
1009866
Contact name
Nina Hou
Contact email
Sponsor organisation
Belite Bio, Inc.
Research summary
Stargardt Disease (STGD1) is the most common form of inherited macular dystrophy. It causes severe and irreversible loss of visual acuity in childhood or adolescence, gradually leading to legally-defined blindness by the first or second decade of life. STGD1 is diagnosed in both children and adults, however, disease progression in childhood-onset STGD1 has been reported to be more rapid and severe, likely due to more severe mutations in a gene known as ABCA4. Because there are no approved treatments for any form of STGD1, it remains a significant area of unmet need. This study is being conducted with participants who have been diagnosed with STGD1 during childhood or adolescence, with at least one mutation in the ABCA4 gene, in order to evaluate safety, tolerability and efficacy of a medication called tinlarebant in early-stage, progressive retinal disease.
The ABCA4 gene encodes a protein (ABCR) which removes a substance called retinaldehyde from the retina during periods of light exposure. If the ABCA4 gene is mutated and therefore cannot encode ABCR protein, then retinaldehyde accumulates in a part of the eye called the retinal pigment epithelium (RPE) and becomes toxic. This build-up of toxicity causes metabolic changes that compromise the RPE’s ability to digest lipofuscin (the “wear-and-tear” pigment), therefore leading to accelerated accumulation of lipofuscin and progressive degeneration of the RPE and photoreceptor cells in the macular region of the eye. Tinlarebant is a retinol binding protein (RBP4) antagonist, meaning it blocks its action. RBP4 is an essential component of the lipofuscin generation cycle, therefore tinlarebant may be able to induce a reduction in RBP4 concentrations, and therefore halt the progression of STGD1.
The total study duration from screening through to follow-up is 26 months, with participants returning to the study site every 3 months for check-ups.
REC name
Wales REC 3
REC reference
24/WA/0134
Date of REC Opinion
14 Jun 2024
REC opinion
Further Information Favourable Opinion