- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05244304
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
- Name: Belitebio - Clinical Trial
- Phone Number: +886 972 080 097
- Email: clinicaltrial@belitebio.com
Study Locations
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South Brisbane, Australia
- Belite Study Site
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New South Wales
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Westmead, New South Wales, Australia
- Belite Study Site
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Victoria
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East Melbourne, Victoria, Australia
- Belite Study Site
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Gent, Belgium
- Belite Study Site
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Leuven, Belgium
- Belite Study Site
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Beijing, China
- Belite Study Site
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Shanghai, China
- Belite Study Site
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Paris, France
- Belite Study Site
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Bonn, Germany
- Belite Study Site
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Gießen, Germany
- Belite Study Site
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Tübingen, Germany
- Belite Study Site
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Kowloon, Hong Kong
- Belite Study Site
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Amsterdam, Netherlands
- Belite Study Site
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Nijmegen, Netherlands
- Belite Study Site
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Basel, Switzerland
- Belite Study Site
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Taipei, Taiwan
- Belite Study Site
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Taoyuan City, Taiwan
- Belite Study Site
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London, United Kingdom
- Belite Study Site
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Southampton, United Kingdom
- Belite Study Site
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California
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Palo Alto, California, United States, 94303
- Belite Study Site
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Florida
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Gainesville, Florida, United States, 32607
- Belite Study Site
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Minnesota
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Minneapolis, Minnesota, United States, 55435
- Belite Study Site
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New York
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New York, New York, United States, 10032
- Belite Study Site
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Utah
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Salt Lake City, Utah, United States, 84132
- Belite Study Site
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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 |
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Experimental: Tinlarebant
5 mg tablet taken orally once a day
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Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
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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.
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Not active drug
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure change in plasma concentration of RBP4 levels (μM) from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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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
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Baseline thru month 24
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To assess the systemic and ocular safety and tolerability of tinlarebant
Time Frame: Baseline thru month 24
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Frequency, duration, and severity of AEs
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Baseline thru month 24
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Other Outcome Measures
Outcome Measure |
Time Frame |
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To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure change in quantitative autofluorescence (qAF) level from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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To measure change in retinal sensitivity by microperimetry from baseline
Time Frame: Baseline thru month 24
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Baseline thru month 24
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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
Additional Relevant MeSH Terms
Other Study ID Numbers
- LBS-008-CT03
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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