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

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.

Study Overview

• Status: Completed
• Conditions: Stargardt Disease
• Intervention / Treatment: Drug: tinlarebant

• Study Type: Interventional
• Enrollment (Actual): 13
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • Sydney Children's Hospitals Network
  • Western Australia
    • Perth, Western Australia, Australia, 6009
      • Lions Eye Institute
• Taiwan
  • Taipei, Taiwan, 100
    • National Taiwan University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 14 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Major 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: tinlarebant; Daily, oral administration of one tinlarebant.	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.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: RBP4 Pty Ltd
• Collaborators: Belite Bio, Inc

Study record dates

Study Major Dates

• Study Start (Actual): March 12, 2021
• Primary Completion (Actual): August 15, 2023
• Study Completion (Actual): August 15, 2023

Study Registration Dates

• First Submitted: January 26, 2022
• First Submitted That Met QC Criteria: March 2, 2022
• First Posted (Actual): March 4, 2022

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Macular Degeneration; Stargardt Disease
• Other Study ID Numbers: LBS-008-CT02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes