Clinical trial NCT06388083: A Phase 2/3 Study to Evaluate the Efficacy and Safety 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: STGD1
Stargardt Disease 1
Lead sponsor: Belite Bio, Inc
Sponsor class: INDUSTRY
First posted: 2024-04-29
Enrollment: 60
Sex: ALL
Minimum age (years): 12
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
timeFrame: From baseline to Month 24
Secondary outcomes:
measure: To measure the annualized rate of change in total area of atrophy
timeFrame: From baseline to Month 24
measure: Change in BCVA measured by the ETDRS method
timeFrame: From baseline to Month 24
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Belite Study Site
state: Arizona
city: La Jolla
country: United States
facility: Belite Study Site
state: California
city: San Francisco
country: United States
facility: Belite Study Site
state: California
city: Miami
country: United States
facility: Belite Study Site
state: Florida
city: Boston
country: United States
facility: Belite Study Site
state: Massachusetts
city: Rochester
country: United States
facility: Belite Study Site
state: Minnesota
city: Westbury
country: United States
facility: Belite Study Site
state: New York
city: Durham
country: United States
facility: Belite Study Site
state: North Carolina
city: Philadelphia
country: United States
facility: Belite Study Site
state: Pennsylvania
city: Dallas
country: United States
facility: Belite Study Site
state: Texas
city: Kobe
country: Japan
facility: Belite Study Site
city: Kyoto
country: Japan
facility: Belite Study Site
city: Tokyo
country: Japan
facility: Belite Study Site
city: London
country: United Kingdom
facility: Bellite Study Site
Study officials:
References: