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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)

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ClinicalTrials.gov Identifier: NCT05244304
Recruitment Status : Active, not recruiting
First Posted : February 17, 2022
Last Update Posted : November 9, 2023
Sponsor:
Information provided by (Responsible Party):
Belite Bio, Inc

Study Description
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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

Condition or disease Intervention/treatment Phase
Stargardt Disease 1 Drug: Tinlarebant Drug: Placebo Phase 3

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 104 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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: Double (Participant, Investigator)
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)
Primary Purpose: Treatment
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
Actual Study Start Date : March 28, 2022
Estimated Primary Completion Date : August 2025
Estimated Study Completion Date : October 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Tinlarebant
5 mg tablet taken orally once a day
 Drug: Tinlarebant
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.
 Drug: Placebo
Not active drug


Outcome Measures
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Primary Outcome Measures :
  1. 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 Measures :
  1. To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline [ Time Frame: Baseline thru month 24 ]
  2. To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline [ Time Frame: Baseline thru month 24 ]
  3. To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline [ Time Frame: Baseline thru month 24 ]
  4. To measure change in plasma concentration of RBP4 levels (μM) from baseline [ Time Frame: Baseline thru month 24 ]
  5. 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 ]
  6. To assess the systemic and ocular safety and tolerability of tinlarebant [ Time Frame: Baseline thru month 24 ]
    Frequency, duration, and severity of AEs


Other Outcome Measures:
  1. To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline [ Time Frame: Baseline thru month 24 ]
  2. To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline [ Time Frame: Baseline thru month 24 ]
  3. To measure change in quantitative autofluorescence (qAF) level from baseline [ Time Frame: Baseline thru month 24 ]
  4. To measure change in retinal sensitivity by microperimetry from baseline [ Time Frame: Baseline thru month 24 ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05244304


Locations
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United States, California
Belite Study Site
Palo Alto, California, United States, 94303
United States, Florida
Belite Study Site
Gainesville, Florida, United States, 32607
United States, Minnesota
Belite Study Site
Minneapolis, Minnesota, United States, 55435
United States, New York
Belite Study Site
New York, New York, United States, 10032
United States, Utah
Belite Study Site
Salt Lake City, Utah, United States, 84132
Australia, New South Wales
Belite Study Site
Westmead, New South Wales, Australia
Australia, Victoria
Belite Study Site
East Melbourne, Victoria, Australia
Australia
Belite Study Site
South Brisbane, Australia
Belgium
Belite Study Site
Gent, Belgium
Belite Study Site
Leuven, Belgium
China
Belite Study Site
Beijing, China
Belite Study Site
Shanghai, China
France
Belite Study Site
Paris, France
Germany
Belite Study Site
Bonn, Germany
Belite Study Site
Gießen, Germany
Belite Study Site
Tübingen, Germany
Hong Kong
Belite Study Site
Kowloon, Hong Kong
Netherlands
Belite Study Site
Amsterdam, Netherlands
Belite Study Site
Nijmegen, Netherlands
Switzerland
Belite Study Site
Basel, Switzerland
Taiwan
Belite Study Site
Taipei, Taiwan
Belite Study Site
Taoyuan City, Taiwan
United Kingdom
Belite Study Site
London, United Kingdom
Belite Study Site
Southampton, United Kingdom
Sponsors and Collaborators
Belite Bio, Inc
More Information
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Responsible Party: Belite Bio, Inc
ClinicalTrials.gov Identifier: NCT05244304    
Other Study ID Numbers: LBS-008-CT03
First Posted: February 17, 2022    Key Record Dates
Last Update Posted: November 9, 2023
Last Verified: November 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Eye Diseases, Hereditary
Eye Diseases
 Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn