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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Belite Bio Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Decision date (initial)

2026-04-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Belite Bio

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To evaluate long-term systemic and ocular safety and tolerability of tinlarebant

Secondary objectives 3

1. To measure the change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline to 36 months, or until marketing authorization is obtained.
2. To measure the change in lesion size (questionably decreased autofluorescence, QDAF) by FAF photography from baseline to 36 months, or until marketing authorization is obtained.
3. To determine the total area of decreased autofluorescence (DAF; the sum of DDAF and QDAF) by FAF photography from baseline to 36 months, or until marketing authorization is obtained.

Conditions and MedDRA coding

Stargardt Disease

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 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.

Exclusion criteria 11

1. Ocular surgery in the study eye in the previous 3 months.
2. 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.
3. 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.
4. Presence of life-threatening disease(s), including current treatment for malignancies.
5. Current alcohol or other substance abuse.
6. Alanine transaminase/aspartate aminotransferase (ALT/AST) > 2.5× the Upper Limit of Normal (ULN) based on last available report.
7. Renal insufficiency, as defined by an eGFR (Bedside Schwartz) < 30 mL/min/1.73m2 based on last available report.
8. 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.
9. 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).
10. Unwillingness or inability to provide signed informed consent prior to participation in any study-related procedures.
11. In the opinion of the Investigator, the subject is not suitable for entry into the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Change in DDAF (Definitely decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography
2. Change in QDAF (Questionably decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography
3. Change in DAF (Decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Belite Bio Inc.

Sponsor organisation

Belite Bio Inc.

Address

Ugland House, P. O. Box 309 P. O. Box 309

City

Grand Cayman

Postcode

KY1-1104

Country

Cayman Islands

Scientific contact point

Organisation

Belite Bio Inc.

Contact name

Chief Medical Officer

Public contact point

Organisation

Belite Bio Inc.

Contact name

Clinical Project Manager

Locations

3 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 29 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications