Clinical trial • Phase III • Ophthalmology
Tinlarebant for Stargardt disease
Phase III trial of Tinlarebant for Stargardt disease. open-label, none/not specified-controlled. 60 participants.
Overview
- Trial Therapeutic Area
- Ophthalmology
- Trial Disease
- Stargardt disease
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 28-11-2025
- First CTIS Authorization Date
- 07-04-2026
Trial design
open-label, none/not specified-controlled Phase III trial across 3 sites in Belgium, France, Netherlands.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 60
- Trial Duration For Participant
- 1095
Eligibility
Recruits 60 paediatric patients.
- Pregnancy Exclusion
- 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.
- Vulnerable Population
- Vulnerable populations are selected for this trial (isVulnerablePopulationSelected = true). The dossier includes assent forms and parent/guardian informed consent forms (e.g. 'L1_SIS and ICF assent form', 'L1_SIS and ICF Main Parent' documents), indicating inclusion of minors where parental/guardian consent plus participant assent is required. Additionally, the exclusion criteria state that unwillingness or inability to provide signed informed consent excludes participation.
Inclusion criteria
- {"criterion_text":"- 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
- {"criterion_text":"- Ocular surgery in the study eye in the previous 3 months.\n- Unwillingness or inability to provide signed informed consent prior to participation in any study-related procedures.\n- In the opinion of the Investigator, the subject is not suitable for entry into the study.\n- 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.\n- 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.\n- Presence of life-threatening disease(s), including current treatment for malignancies.\n- Current alcohol or other substance abuse.\n- Alanine transaminase/aspartate aminotransferase (ALT/AST) > 2.5× the Upper Limit of Normal (ULN) based on last available report.\n- Renal insufficiency, as defined by an eGFR (Bedside Schwartz) < 30 mL/min/1.73m2 based on last available report.\n- 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.\n- 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)."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change in DDAF (Definitely decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography","definition_or_measurement_approach":"Assessed by fundus autofluorescence (FAF) photography; change measured from baseline to 36 months (longitudinal measurement of DDAF area)."}
- {"endpoint_text":"- Change in QDAF (Questionably decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography","definition_or_measurement_approach":"Assessed by fundus autofluorescence (FAF) photography; change measured from baseline to 36 months (longitudinal measurement of QDAF area)."}
- {"endpoint_text":"- Change in DAF (Decreased autofluorescence) assessed by FAF (Fundus autofluorescence) photography","definition_or_measurement_approach":"Assessed by fundus autofluorescence (FAF) photography; DAF = sum of DDAF and QDAF, change measured from baseline to 36 months."}
Recruitment
- Planned Sample Size
- 60
- Recruitment Window Months
- 36
- Consent Approach
- Informed consent is required from participants prior to any study procedures; the dossier includes specific subject information and informed consent forms and assent forms (e.g. 'L1_SIS and ICF main ICF', 'L1_SIS and ICF Main Parent', 'L1_SIS and ICF assent form'). Parent/guardian consent is used for minors with assent forms for the minor participants. Documents are available in multiple languages for country-specific use (filenames indicate English, French, Dutch versions). Exclusion includes inability or unwillingness to provide signed informed consent.
Geography
- Total Number Of Sites
- 3
- Total Number Of Participants
- 6
Belgium
- Earliest CTIS Part Ii Submission Date
- 19-03-2026
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 20
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- ophthalmology
- Principal Investigator Name
- Bart Leroy
- Principal Investigator Email
- bart.leroy@ugent.be
- Contact Person Name
- Bart Leroy
- Contact Person Email
- bart.leroy@ugent.be
- Number Of Participants
- 3
France
- Earliest CTIS Part Ii Submission Date
- 25-03-2026
- Latest Decision Or Authorization Date
- 08-04-2026
- Processing Time Days
- 14
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Centre Hospitalier National d'Ophtalmologie des Quize-Vingts
- Department Name
- Ophtalmology
- Principal Investigator Name
- Isabelle Audo
- Principal Investigator Email
- isabelle.audo@inserm.fr
- Contact Person Name
- Isabelle Audo
- Contact Person Email
- isabelle.audo@inserm.fr
- Number Of Participants
- 2
Netherlands
- Earliest CTIS Part Ii Submission Date
- 16-03-2026
- Latest Decision Or Authorization Date
- 07-04-2026
- Processing Time Days
- 22
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Ophtalmology
- Principal Investigator Name
- Carel Hoyng
- Principal Investigator Email
- carel.hoyng@radboudumc.nl
- Contact Person Name
- Carel Hoyng
- Contact Person Email
- carel.hoyng@radboudumc.nl
- Number Of Participants
- 1
Sponsor
Primary sponsor
- Full Name
- Belite Bio Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Cayman Islands
Investigational products
- Investigational Product Name
- LBS-008
- Active Substance
- Tinlarebant
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- ORAL USE
- Authorisation Status
- prodAuthStatus: 1
- Orphan Designation
- Yes
- Maximum Dose
- 5 mg (max daily dose amount: 5 mg)
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