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
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
104
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
Not active drug
Belite Study Site
Palo Alto, California, United States
Belite Study Site
Minneapolis, Minnesota, United States
Belite Study Site
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time frame: Baseline thru month 24
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time frame: Baseline thru month 24
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time frame: Baseline thru month 24
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Time frame: Baseline thru month 24
To measure change in plasma concentration of RBP4 levels (μM) from baseline
Time frame: 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
Time frame: Baseline thru month 24
To assess the systemic and ocular safety and tolerability of tinlarebant
Frequency, duration, and severity of AEs
Time frame: Baseline thru month 24
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Salt Lake City, Utah, United States
Belite Study Site
Westmead, New South Wales, Australia
Belite Study Site
East Melbourne, Victoria, Australia
Belite Study Site
South Brisbane, Australia
Belite Study Site
Ghent, Belgium
Belite Study Site
Beijing, China
Belite Study Site
Shanghai, China
Belite Study Site
Paris, France
...and 9 more locations