Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease: An Open-Label, Single-Arm, 3-Year Extension Study

Trial ID: 2025-522460-33-00
Protocol: LBS-008-CT08
Sponsor: Belite Bio Inc.

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Diseases & Conditions

Stargardt's disease

Objectives

The primary objective of this study is to evaluate the long-term systemic and ocular safety and tolerability of tinlarebant in subjects with Stargardt disease. The secondary objectives involve the assessment of retinal changes via fundus autofluorescence (FAF) photography from baseline to 36 months, including:

Measurement of the change in size of atrophic lesions, specifically definitely decreased autofluorescence (DDAF).
Measurement of the change in size of questionably decreased autofluorescence (QDAF) lesions.
Determination of the total area of decreased autofluorescence (DAF), defined as the sum of DDAF and QDAF.

Participants

The study population consists of 60 patients diagnosed with Stargardt disease. The cohort includes both male and female participants. The sponsor did not provide specific information regarding the age range or lifestyle considerations. Selection is based on the completion of a 24-month treatment period in either the LBS-008-CT02 or LBS-008-CT03 trial, including the fulfillment of all required end-of-treatment assessments.

Plans and Procedures

This open-label, single-arm, phase III extension study is designed to evaluate the long-term systemic and ocular safety and tolerability of tinlarebant in subjects diagnosed with Stargardt disease. The research methodology involves the continued administration of LBS-008, an oral tablet, for a duration of 3 years. Eligibility is restricted to participants who have successfully completed a 24-month treatment period in either the LBS-008-CT02 or LBS-008-CT03 trials and have fulfilled all requirements of the end-of-treatment visit. The primary endpoints include assessing changes in fundus autofluorescence (FAF) photography, specifically regarding definitely decreased autofluorescence (DDAF), questionably decreased autofluorescence (QDAF), and decreased autofluorescence (DAF). The study period is expected to conclude by April 2029.

Treatment

The experimental medication consists of tinlarebant, an orphan drug administered as LBS-008. This substance is provided in a tablet pharmaceutical form for oral use at a dosage of 5 mg. The study is designed to evaluate the long-term systemic and ocular safety and tolerability of this agent in subjects diagnosed with Stargardt disease.

Efficacy

The efficacy assessment focuses on evaluating changes in specific retinal characteristics associated with Stargardt disease. The primary endpoints consist of the change in definitely decreased autofluorescence (DDAF), questionably decreased autofluorescence (QDAF), and decreased autofluorescence (DAF).

Measurements for these parameters will be performed using fundus autofluorescence (FAF) photography.

Inclusion and Exclusion Criteria

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Inclusion Criteria

Subject has completed 24-month treatment in LBS-008-CT02 or LBS-008-CT03 trial and has also completed the tests and assessments required end-of-treatment visit.

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Exclusion Criteria

Ocular surgery in the study eye in the previous 3 months.
Use of prescription medications such as Isotretinoin (13-cis-retinoic acid) or other retinol modulators or derivatives which may impact the effect of the study drug.
Use of any known drugs or supplements that are inhibitors/inducers of cytochrome P450 (CYP) enzymes (e.g., rifampin, barbiturates, phenothiazines, cimetidine, carbamazepine, St. John’s wort) within 30 days of study drug administration or consumption of foods that are inhibitors/inducers of CYP3A4 (e.g., grapefruit, bitter orange [Seville orange], pomegranate, or star fruit) within 48 hours of study drug administration, and that, in the Investigator’s judgement, may impact subjects’ safety or the validity of the study results.
Presence of life-threatening disease(s), including current treatment for malignancies.
Current alcohol or other substance abuse.
Alanine transaminase/aspartate aminotransferase (ALT/AST) > 2.5× the Upper Limit of Normal (ULN) based on last available report.
Renal insufficiency, as defined by an eGFR (Bedside Schwartz) < 30 mL/min/1.73m2 based on last available report.
Pregnant or nursing females and females of childbearing potential who are unwilling or unable to use an acceptable method of contraception (or abstinence). Females must have a negative pregnancy during the treatment period.
Male subject who does not agree that female spouse/partner will use adequate contraception (i.e., condoms) or be of non-childbearing potential (i.e., surgically sterile).
Unwillingness or inability to provide signed informed consent prior to participation in any study-related procedures.
In the opinion of the Investigator, the subject is not suitable for entry into the study.