Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 (DRAGON)

November 8, 2023 updated by: Belite Bio, Inc

Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects

The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

Study Type

Interventional

Enrollment (Actual)

104

Phase

  • 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

      • South Brisbane, Australia
        • Belite Study Site
    • New South Wales
      • Westmead, New South Wales, Australia
        • Belite Study Site
    • Victoria
      • East Melbourne, Victoria, Australia
        • Belite Study Site
      • Gent, Belgium
        • Belite Study Site
      • Leuven, Belgium
        • Belite Study Site
      • Beijing, China
        • Belite Study Site
      • Shanghai, China
        • Belite Study Site
      • Paris, France
        • Belite Study Site
      • Bonn, Germany
        • Belite Study Site
      • Gießen, Germany
        • Belite Study Site
      • Tübingen, Germany
        • Belite Study Site
      • Kowloon, Hong Kong
        • Belite Study Site
      • Amsterdam, Netherlands
        • Belite Study Site
      • Nijmegen, Netherlands
        • Belite Study Site
      • Basel, Switzerland
        • Belite Study Site
      • Taipei, Taiwan
        • Belite Study Site
      • Taoyuan City, Taiwan
        • Belite Study Site
      • London, United Kingdom
        • Belite Study Site
      • Southampton, United Kingdom
        • Belite Study Site
    • California
      • Palo Alto, California, United States, 94303
        • Belite Study Site
    • Florida
      • Gainesville, Florida, United States, 32607
        • Belite Study Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55435
        • Belite Study Site
    • New York
      • New York, New York, United States, 10032
        • Belite Study Site
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • 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

12 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female subjects 12 to 20 years old, inclusive.
  • Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
  • Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
  • Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
  • Subject agrees to comply with all protocol requirements.

Exclusion Criteria:

  • Any ocular disease other than Stargardt (STGD1) at baseline 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.
  • Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
  • Any prior gene therapy.
  • Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).

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: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tinlarebant
5 mg tablet taken orally once a day
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
Placebo Comparator: Placebo
Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.
Not active drug

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure change in plasma concentration of RBP4 levels (μM) from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To assess the systemic and ocular safety and tolerability of tinlarebant
Time Frame: Baseline thru month 24
Frequency, duration, and severity of AEs
Baseline thru month 24

Other Outcome Measures

Outcome Measure
Time Frame
To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure change in quantitative autofluorescence (qAF) level from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24
To measure change in retinal sensitivity by microperimetry from baseline
Time Frame: Baseline thru month 24
Baseline thru month 24

Collaborators and Investigators

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

Sponsor

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 (Actual)

March 28, 2022

Primary Completion (Estimated)

August 1, 2025

Study Completion (Estimated)

October 1, 2025

Study Registration Dates

First Submitted

January 20, 2022

First Submitted That Met QC Criteria

February 16, 2022

First Posted (Actual)

February 17, 2022

Study Record Updates

Last Update Posted (Estimated)

November 9, 2023

Last Update Submitted That Met QC Criteria

November 8, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

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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