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

Completed

• Conditions: Stargardt Disease
• Interventions: Drug: tinlarebant

• Registration Number: NCT05266014
• Lead Sponsor: RBP4 Pty Ltd

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-03-12
• Study First Submitted: 2022-01-26
• Study First Submitted QC: 2022-03-02
• Study First Posted: 2022-03-04
• Primary Completion: 2023-08-15
• Study Completion: 2023-08-15
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-10
• Last Update Posted: 2024-04-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

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

Major

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
tinlarebant	tinlarebant	Daily, oral administration of one tinlarebant.

Primary Outcome Measures

Name	Time	Method
To evaluate systemic and ocular safety and tolerability of tinlarebant.	From baseline to 24 months	To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
The optimal dose for Phase 2.	Up to 24 months	To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Secondary Outcome Measures

Name	Time	Method
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.	Up to 24 months
Half-life (t1/2) of tinlarebant in plasma.	Up to 24 months
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.	Up to 24 months
Time to minimal plasma RBP4 level (Tmin)	Up to 24 months
Change in atrophic lesion size.	From baseline to 24 months.
Minimum concentration of RBP4 (Cmin)	Up to 24 months

Trial Locations

Locations (3)

Sydney Children's Hospitals Network; 🇦🇺 Westmead, New South Wales, Australia

Lions Eye Institute; 🇦🇺 Perth, Western Australia, Australia

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan