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 Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05266014
• Other study ID #: LBS-008-CT02
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: March 12, 2021
• Est. completion date: August 15, 2023

Study information

• Verified date: April 2024
• Source: RBP4 Pty Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: August 15, 2023
• Est. primary completion date: August 15, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt Disease

Intervention

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.

Locations

Country	Name	City	State
Australia	Lions Eye Institute	Perth	Western Australia
Australia	Sydney Children's Hospitals Network	Westmead	New South Wales
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (2)

Lead Sponsor	Collaborator
RBP4 Pty Ltd	Belite Bio, Inc

Countries where clinical trial is conducted

Australia,  Taiwan, 

Outcome

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