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Completed NCT05244304

Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease

Conditions: Stargardt Disease 1

Sex: All
Ages: 12 Years – 20 Years
Healthy volunteers: No
Phase: PHASE3
Enrollment: 104
Sponsor: Belite Bio, Inc

Location: Belite Study Site Palo Alto California

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

Eligibility Criteria

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).

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT05244304). StuddyBuddy aggregates publicly available trial information.