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

NCT06388083 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: LBS-008-CT07
• Study Type: interventional
• Target: 60
• Locations: 3 countries
• Sites: 18

Brief Summary

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

Conditions

STGD1; Stargardt Disease 1

Outcome Measures

Primary Outcomes (1)

• To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy

  From baseline to Month 24

Secondary Outcomes (2)

• To measure the annualized rate of change in total area of atrophy

  From baseline to Month 24

• Change in BCVA measured by the ETDRS method

  From baseline to Month 24

Study Arms (2)

LBS-008, Tinlarebant

EXPERIMENTAL

5 mg tablet taken orally once a day

Drug: Tinlarebant

Placebo

PLACEBO COMPARATOR

Placebo tablets for tinlarebant 5 mg prepared similarly

Drug: Placebo

Interventions

Tinlarebant DRUG

5 mg tablet

Also known as: LBS-008

LBS-008, Tinlarebant

Placebo DRUG

Placebo tablets

Placebo

Eligibility Criteria

• Age: 12 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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 may not qualify 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.

Sponsors & Collaborators

• Belite Bio, Inc: lead

Study Sites (18)

Belite Study Site - US08

Phoenix, Arizona, 85020, United States

Location

Belite Study Site - US05

La Jolla, California, 92093, United States

Location

Belite Study Site - US09

San Francisco, California, 94158, United States

Location

Belite Study Site - US06

Miami, Florida, 33136, United States

Location

Belite Study Site - US01

Boston, Massachusetts, 02115, United States

Location

Belite Study Site - US11

Edina, Minnesota, 55435, United States

Location

Belite Study Site - US04

Rochester, Minnesota, 55905, United States

Location

Belite Study Site - US14

New York, New York, 10032, United States

Location

Belite Study Site - US10

Westbury, New York, 11590, United States

Location

Belite Study Site - US02

Durham, North Carolina, 27705, United States

Location

Belite Study Site - US07

Philadelphia, Pennsylvania, 19107, United States

Location

Belite Study Site - US13

Austin, Texas, 78705, United States

Location

Belite Study Site - US03

Dallas, Texas, 75231, United States

Location

Belite Study Site - US12

The Woodlands, Texas, 77384, United States

Location

Belite Study Site

Kobe, Japan

Location

Belite Study Site

Kyoto, Japan

Location

Belite Study Site

Tokyo, Japan

Location

UK01 Belite Study Site

London, United Kingdom

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2024
• First Posted: April 29, 2024
• Study Start: July 31, 2024
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2028
• Last Updated: March 12, 2026

Record last verified: 2026-03

Locations

GB (1); JP (3); US (14)