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

NCT05266014 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: LBS-008-CT02
• Study Type: interventional
• Target: 13
• Locations: 2 countries
• Sites: 3

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (6)

• 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

Study Arms (1)

tinlarebant

EXPERIMENTAL

Daily, oral administration of one tinlarebant.

Drug: tinlarebant

Interventions

tinlarebant 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.

tinlarebant

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• RBP4 Pty Ltd: lead
• Belite Bio, Inc: collaborator

Study Sites (3)

Sydney Children's Hospitals Network

Westmead, New South Wales, 2145, Australia

Location

Lions Eye Institute

Perth, Western Australia, 6009, Australia

Location

National Taiwan University Hospital

Taipei, 100, Taiwan

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2022
• First Posted: March 4, 2022
• Study Start: March 12, 2021
• Primary Completion: August 15, 2023
• Study Completion: August 15, 2023
• Last Updated: April 12, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2); TW (1)