A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease

a study on STGD1 Stargardt Disease

Summary

Eligibility
for people ages 12-20 (full criteria)
Location
at UCSD UCSF
Dates
study started
study ends around

Description

Summary

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

Official Title

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

Details

This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.

Keywords

STGD1, Stargardt Disease 1, Stargardt Disease, Counterfeit Drugs, Tinlarebant, LBS-008, Tinlarebant

Eligibility

You can join if…

Open to people ages 12-20

  • 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

You CAN'T join if...

  • 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.

Locations

  • Belite Study Site - US05 accepting new patients
    La Jolla 5363943 California 5332921 92093 United States
  • Belite Study Site - US09 not yet accepting patients
    San Francisco 5391959 California 5332921 94158 United States
  • Belite Study Site - US08 accepting new patients
    Phoenix 5308655 Arizona 5551752 85020 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Belite Bio, Inc
ID
NCT06388083
Phase
Phase 2/3 research study
Study Type
Interventional
Participants
Expecting 60 study participants
Last Updated