Stargardt Disease
Conditions
Interventions
Product Name: Tinlarebant
Product Code: LBS-008, BPN-14967
Pharmaceutical Form: Tablet
INN or Proposed INN: Tinlarebant
CAS Number: NA
Current Sponsor code: LBS-008, BPN-14967
Concentration unit: mg m
Sponsors
Belite Bio, Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female subjects 12 to 18 years old, inclusive. 2. Subject must have clinically diagnosed STGD1 with at least 1 mutation identified in the ABCA4 gene. 3. Ability to adequately examine the fundus of the study eye at enrollment. a. The study eye will be the eye that meets all inclusion and exclusion criteria. b. If both eyes meet all inclusion and exclusion criteria, the eye with the smaller lesion will be selected to be the study eye. c. If both eyes meet all inclusion and exclusion criteria and have lesions of equal size, then the eye with the better BCVA will be selected to be the study eye. d. If both eyes meet all inclusion and exclusion criteria, have lesions of equal size, and have equal BCVA, the default will be the right eye. 4. Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2) as imaged by FAF (DDAF) in the study eye and confirmed by the central reading center (Fleckenstein et al, 2011; Sunness et al, 2007). The lesion, or at least 1 focal lesion if multiple lesions exist (multifocal atrophic lesions), must be in the macular region and greater than 0.02 disc areas (0.05 mm2). 5. Subjects must present with BCVA of 20/200 or better for the study eye based on ETDRS letter score. No minimum VA is required in the fellow eye. 6. Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures. 7. Subject agrees to comply with all protocol requirements. Are the trial subjects under 18? yes Number of subjects for this age range: 60 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any ocular disease other than STGD1 at screening that, in the opinion of the investigator, would complicate assessment of a treatment effect. 2. History of ocular surgery in the study eye in the last 3 months. 3. Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter. 4. Any prior gene therapy. 5. Vitamin A deficiency as defined based upon plasma values less than 20 µg/dL (=0.7 µmol/L). 6. Use of medications such as isotretinoin (13-cis-retinoic acid) or other retinol modulators or derivatives that may impact the effect of the study drug in the last 2 weeks or within the washout period of the medication, whichever is longer, before beginning study treatment administration. 7. Unwilling to discontinue vitamin A or beta-carotene supplement use. 8. Use of any known drugs or supplements that are moderate or strong inhibitors/inducers of cytochrome P450 (CYP) enzymes (eg, rifampin, barbiturates, phenothiazines, cimetidine, carbamazepine, St. John’s wort) within 30 days of study drug administration or consumption of foods that are moderate or strong inhibitors/inducers of CYP enzymes (eg, grapefruit, pomegranate, star fruit, bitter orange [Seville orange]) within 48 hours of study drug administration, and that, in the investigator’s judgement, may impact subject safety or the validity of the study results. 9. Presence of any life-threatening disease(s), including current treatment for malignancies. 10. Alanine transaminase/aspartate aminotransferase >2.5 × the upper limit of normal at screening. 11. Renal insufficiency, as defined by an estimated glomerular filtration rate (Bedside Schwartz) <30 mL/min/1.73 m2at screening. 12. Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of contraception (or abstinence). Females of childbearing age must have a negative pregnancy test prior to randomization (Appendix 13.5). 13. Male subjects who do not agree that female spouses/partners will use adequate contraception (eg, condoms) or be of nonchildbearing potential (ie, surgically sterile) (Appendix 13.5). 14. Unwilling or unable to provide signed informed consent/assent. 15. In the opinion of the investigator, the subject is not suitable for entry into the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1;Secondary Objective: To assesses the efficacy of tinlarebant in adolescent subjects with STGD1 for secondary endpoints (Secondary efficacy endpoints will be assessed at Month 24.) To evaluate the pharmacodynamics (PD) of tinlarebant in adolescent subjects with STGD1 To assess systemic and ocular safety and tolerability of tinlarebant;Primary end point(s): Rate of change from baseline in aggregate area of atrophy (definitely decreased autofluorescence [DDAF]) assessed by FAF photography.;Timepoint(s) of evaluation of this end point: 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in retinal thickness and morphology by SDOCT from baseline • Change in retinal sensitivity by microperimetry from baseline • Change in BCVA measured by the ETDRS method from baseline • Change in RBP4 levels from baseline • Correlation between the change in RBP4 level and the rate of lesion size growth from baseline • Physical examination, vital signs measurement, ECG, ophthalmic examination, clinical laboratory tests (including serum chemistry and hematology panels, urinalysis, and pregnancy tests on all female subjects), retinol chemistries (plasma retinol, plasma RBP4), visual function questionnaire, measurement of intraocular pressure (IOP), dark adaptation test, dilated funduscopy, contrast sensitivity, assessment of adverse events (AEs), and monitoring of concomitant medications. ;Timepoint(s) of evaluation of this end point: Evaluation on the following months: 1, 4, 7, 10, 13, 16, 19 and 22 | — |
Countries
Australia, Belgium, China, France, Germany, Hong Kong, Switzerland, Taiwan, United Kingdom, United States
Contacts
Public ContactNathan Mata
Belite Bio, Inc
Outcome results
None listed