Skip to main content
OpenTrials
Completed

NCT Number: NCT05266014

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

12 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. To evaluate systemic and ocular safety and tolerability of tinlarebant.

    Time frame: 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.

  2. The optimal dose for Phase 2.

    Time frame: Up to 24 months

    To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Secondary outcomes

  1. Change in atrophic lesion size.

    Time frame: From baseline to 24 months.

  2. Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.

    Time frame: Up to 24 months

  3. Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.

    Time frame: Up to 24 months

  4. Half-life (t1/2) of tinlarebant in plasma.

    Time frame: Up to 24 months

  5. Time to minimal plasma RBP4 level (Tmin)

    Time frame: Up to 24 months

  6. Minimum concentration of RBP4 (Cmin)

    Time frame: Up to 24 months

Sponsors and collaborators

Lead sponsor

RBP4 Pty Ltd

Industry

Collaborators

  • Belite Bio, Inc

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Mar 4, 2022
Registry last updated
Apr 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.