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

NCT ID: NCT05266014

Last Updated: 2024-04-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-12
• Study Completion Date: 2023-08-15

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

tinlarebant

Daily, oral administration of one tinlarebant.

Group Type: EXPERIMENTAL

tinlarebant

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

Any ocular disease other than Stargardt disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Belite Bio, Inc

INDUSTRY

Sponsor Role: collaborator

RBP4 Pty Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Sydney Children's Hospitals Network

Westmead, New South Wales, Australia

Lions Eye Institute

Perth, Western Australia, Australia

National Taiwan University Hospital

Taipei, , Taiwan

Countries

Australia; Taiwan

Other Identifiers

LBS-008-CT02

Identifier Type: -

Identifier Source: org_study_id