NCT05266014

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Phase 1/2, Open-Label, Dose-Finding Followed by 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: RBP4 Pty Ltd · Industry
• Sex: All
• Age: 12 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis. This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.

Conditions

• Stargardt Disease

Interventions

Type	Name	Description
DRUG	tinlarebant	Phase 1b Portion: tinlarebant will be self-administered orally once daily for 2 cycles, 14 days per cycle. Phase 2 portion: tinlarebant will be self-administered orally once daily for 24 months.

Timeline

• Start date: 2021-03-12
• Primary completion: 2023-08-15
• Completion: 2023-08-15
• First posted: 2022-03-04
• Last updated: 2024-04-12

Locations

3 sites across 2 countries: Australia, Taiwan

Regulatory

• FDA-regulated drug study