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

Who is this study for? Patients with Stargardt Disease
What treatments are being studied? Tinlarebant
Status: Completed
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 18
Healthy Volunteers: f
View:

• Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.

Locations
Other Locations
Australia
Lions Eye Institute
Perth
Sydney Children's Hospitals Network
Westmead
Taiwan
National Taiwan University Hospital
Taipei
Time Frame
Start Date: 2021-03-12
Completion Date: 2023-08-15
Participants
Target number of participants: 13
Treatments
Experimental: tinlarebant
Daily, oral administration of one tinlarebant.
Sponsors
Collaborators: Belite Bio, Inc
Leads: RBP4 Pty Ltd

This content was sourced from clinicaltrials.gov

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