A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

LBS-008-CT07

The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

2024-06-01

2027-06-01

2027-07-01

Not yet recruiting

Early phase I

60

Inclusion Criteria: * Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene. * Subjects must have a defined aggregate atrophic lesion in 1 or both eyes. * Minimum BCVA is required in the study eye Exclusion Criteria: * Any ocular disease other than STGD1 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. * Any prior gene therapy.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 60, 'type': 'ESTIMATED'}}

2024-04-29