Tinlarebant for Stargardt Disease
(DRAGON Trial)

Recruiting in Palo Alto (17 mi)

+50 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Belite Bio, Inc

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a medication called tinlarebant to see if it can slow down eye damage in teenagers with a condition called STGD1. About 90 participants will take tinlarebant for an extended period. The goal is to see if tinlarebant can help prevent their vision from getting worse.

Eligibility Criteria

Adolescents aged 12-20 with Stargardt Disease (STGD1) and a specific gene mutation. They must have certain eye health criteria, like a lesion size within limits and vision of 20/200 or better. Participants need to agree to follow the study rules and provide informed consent.

Inclusion Criteria

I have Stargardt disease with a confirmed ABCA4 gene mutation.

Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score

My eye condition has been confirmed with a specific imaging test.

+8 more

Exclusion Criteria

Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L)

I have no eye conditions other than Stargardt disease that could affect treatment assessment.

I have received gene therapy before.

+2 more

Participant Groups

The trial is testing Tinlarebant's effectiveness in slowing down the growth of lesions in the eyes caused by STGD1 compared to a placebo. The main goal is to see if this drug can help preserve vision in affected adolescents.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: TinlarebantExperimental Treatment1 Intervention

5 mg tablet taken orally once a day

Group II: PlaceboPlacebo Group1 Intervention

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.