Stargardt Disease 1
Conditions
Brief summary
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
Detailed description
Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.
Interventions
DRUGTinlarebant
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
DRUGPlacebo
Not active drug
Sponsors
Belite Bio, Inc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
Eligible subjects will be randomly assigned to begin treatment in 2:1 ratio to receive the study drug (either Tinlarebant 5 mg or matching placebo)
Intervention model description
This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
Eligibility
Sex/Gender
ALL
Age
12 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
* Male or female subjects 12 to 20 years old, inclusive. * Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene. * Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score * 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. * Subject agrees to comply with all protocol requirements.
Exclusion criteria
* Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect. * History of ocular surgery in the study eye in the last 3 months. * Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter. * Any prior gene therapy. * Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline | Baseline thru month 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline | Baseline thru month 24 | — |
| To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline | Baseline thru month 24 | — |
| To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline | Baseline thru month 24 | — |
| To measure change in plasma concentration of RBP4 levels (μM) from baseline | Baseline thru month 24 | — |
| The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline | Baseline thru month 24 | — |
| To assess the systemic and ocular safety and tolerability of tinlarebant | Baseline thru month 24 | Frequency, duration, and severity of AEs |
Countries
Australia, Belgium, China, France, Germany, Hong Kong, Netherlands, Switzerland, Taiwan, United Kingdom, United States
Outcome results
None listed