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

April 26, 2024 updated by: Belite Bio, Inc

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

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

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75231
        • Belite Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: LBS-008, Tinlarebant
5 mg tablet taken orally once a day
Drug: Tinlarebant
5 mg tablet
Other Names:
  • LBS-008
Placebo Comparator: Placebo
Placebo tablets for tinlarebant 5 mg prepared similarly
Drug: Placebo
Placebo tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
Time Frame: From baseline to Month 24
From baseline to Month 24

Secondary Outcome Measures

Outcome Measure
Time Frame
To measure the annualized rate of change in total area of atrophy
Time Frame: From baseline to Month 24
From baseline to Month 24
Change in BCVA measured by the ETDRS method
Time Frame: From baseline to Month 24
From baseline to Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Belite Bio, Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 1, 2024

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

July 1, 2027

Study Registration Dates

First Submitted

April 23, 2024

First Submitted That Met QC Criteria

April 26, 2024

First Posted (Actual)

April 29, 2024

Study Record Updates

Last Update Posted (Actual)

April 29, 2024

Last Update Submitted That Met QC Criteria

April 26, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LBS-008-CT07

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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