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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Tinlarebant | Experimental | 5 mg tablet taken orally once a day |
|
| Placebo | Placebo Comparator | Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tinlarebant | Drug | Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline | Baseline thru month 24 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline | Baseline thru month 24 | |
| To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline | Baseline thru month 24 |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Belite Study Site | Palo Alto | California | 94303 | United States | ||
| Belite Study Site |
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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.
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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)
| Placebo | Drug | Not active drug |
|
| 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 | Frequency, duration, and severity of AEs | Baseline thru month 24 |
| To measure change in quantitative autofluorescence (qAF) level from baseline | Baseline thru month 24 |
| To measure change in retinal sensitivity by microperimetry from baseline | Baseline thru month 24 |
| Minneapolis |
| Minnesota |
| 55435 |
| United States |
| Belite Study Site | Salt Lake City | Utah | 84132 | 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 |
| Belite Study Site | Bonn | Germany |
| Belite Study Site | Tübingen | Germany |
| Belite Study Site | Kowloon | Hong Kong |
| Belite Study Site | Nijmegen | Netherlands |
| Belite Study Site | Basel | Switzerland |
| Belite Study Site | Taipei | Taiwan |
| Belite Study Site | Taoyuan City | Taiwan |
| Belite Study Site | London | United Kingdom |
| Belite Study Site | Southampton | United Kingdom |
| ID | Term |
|---|---|
| D000080362 | Stargardt Disease |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D008268 | Macular Degeneration |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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