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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)

Primary Purpose

Stargardt Disease 1

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Tinlarebant
Placebo
Sponsored by
Belite Bio, Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Stargardt Disease 1

Eligibility Criteria

12 Years - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male or female subjects 12 to 18 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).

Sites / Locations

  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site
  • Belite Study Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Tinlarebant

Placebo

Arm Description

5 mg tablet taken orally once a day

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.

Outcomes

Primary Outcome Measures

To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline

Secondary Outcome Measures

To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
To measure change in plasma concentration of RBP4 levels (μM) from baseline
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
To assess the systemic and ocular safety and tolerability of tinlarebant
Frequency, duration, and severity of AEs

Full Information

First Posted
January 20, 2022
Last Updated
July 31, 2023
Sponsor
Belite Bio, Inc
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1. Study Identification

Unique Protocol Identification Number
NCT05244304
Brief Title
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
Acronym
DRAGON
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
March 28, 2022 (Actual)
Primary Completion Date
August 2025 (Anticipated)
Study Completion Date
October 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Belite Bio, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Stargardt Disease 1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
Masking
ParticipantInvestigator
Masking Description
Eligible subjects will be randomly assigned to begin treatment in 2:1 ratio to receive the study drug (either Tinlarebant 5 mg or matching placebo)
Allocation
Randomized
Enrollment
90 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Tinlarebant
Arm Type
Experimental
Arm Description
5 mg tablet taken orally once a day
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Tinlarebant
Intervention Description
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Not active drug
Primary Outcome Measure Information:
Title
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time Frame
Baseline thru month 24
Secondary Outcome Measure Information:
Title
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame
Baseline thru month 24
Title
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame
Baseline thru month 24
Title
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Time Frame
Baseline thru month 24
Title
To measure change in plasma concentration of RBP4 levels (μM) from baseline
Time Frame
Baseline thru month 24
Title
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
Title
To assess the systemic and ocular safety and tolerability of tinlarebant
Description
Frequency, duration, and severity of AEs
Time Frame
Baseline thru month 24
Other Pre-specified Outcome Measures:
Title
To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline
Time Frame
Baseline thru month 24
Title
To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline
Time Frame
Baseline thru month 24
Title
To measure change in quantitative autofluorescence (qAF) level from baseline
Time Frame
Baseline thru month 24
Title
To measure change in retinal sensitivity by microperimetry from baseline
Time Frame
Baseline thru month 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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).
Facility Information:
Facility Name
Belite Study Site
City
Palo Alto
State/Province
California
ZIP/Postal Code
94303
Country
United States
Facility Name
Belite Study Site
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Belite Study Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55435
Country
United States
Facility Name
Belite Study Site
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Belite Study Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Belite Study Site
City
Westmead
State/Province
New South Wales
Country
Australia
Facility Name
Belite Study Site
City
East Melbourne
State/Province
Victoria
Country
Australia
Facility Name
Belite Study Site
City
South Brisbane
Country
Australia
Facility Name
Belite Study Site
City
Gent
Country
Belgium
Facility Name
Belite Study Site
City
Leuven
Country
Belgium
Facility Name
Belite Study Site
City
Beijing
Country
China
Facility Name
Belite Study Site
City
Shanghai
Country
China
Facility Name
Belite Study Site
City
Paris
Country
France
Facility Name
Belite Study Site
City
Bonn
Country
Germany
Facility Name
Belite Study Site
City
Gießen
Country
Germany
Facility Name
Belite Study Site
City
Tübingen
Country
Germany
Facility Name
Belite Study Site
City
Kowloon
Country
Hong Kong
Facility Name
Belite Study Site
City
Amsterdam
Country
Netherlands
Facility Name
Belite Study Site
City
Nijmegen
Country
Netherlands
Facility Name
Belite Study Site
City
Basel
Country
Switzerland
Facility Name
Belite Study Site
City
Taipei
Country
Taiwan
Facility Name
Belite Study Site
City
Taoyuan City
Country
Taiwan
Facility Name
Belite Study Site
City
London
Country
United Kingdom
Facility Name
Belite Study Site
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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