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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05244304
Registration number
NCT05244304
Ethics application status
Date submitted
20/01/2022
Date registered
17/02/2022
Date last updated
7/06/2024
Titles & IDs
Public title
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
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Scientific 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
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Secondary ID [1]
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LBS-008-CT03
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Universal Trial Number (UTN)
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Trial acronym
DRAGON
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Stargardt Disease 1
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Condition category
Condition code
Eye
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Diseases / disorders of the eye
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Tinlarebant
Treatment: Drugs - Placebo
Experimental: Tinlarebant - 5 mg tablet taken orally once a day
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.
Treatment: Drugs: Tinlarebant
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
Treatment: Drugs: Placebo
Not active drug
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
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Assessment method [1]
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Timepoint [1]
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Baseline thru month 24
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Secondary outcome [1]
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To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
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Assessment method [1]
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Timepoint [1]
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Baseline thru month 24
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Secondary outcome [2]
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To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
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Assessment method [2]
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Timepoint [2]
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Baseline thru month 24
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Secondary outcome [3]
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To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
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Assessment method [3]
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Timepoint [3]
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Baseline thru month 24
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Secondary outcome [4]
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To measure change in plasma concentration of RBP4 levels (µM) from baseline
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Assessment method [4]
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Timepoint [4]
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Baseline thru month 24
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Secondary outcome [5]
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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
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Assessment method [5]
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Timepoint [5]
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Baseline thru month 24
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Secondary outcome [6]
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To assess the systemic and ocular safety and tolerability of tinlarebant
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Assessment method [6]
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Frequency, duration, and severity of AEs
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Timepoint [6]
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Baseline thru month 24
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Eligibility
Key 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.
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Minimum age
12
Years
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Maximum age
20
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/03/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/10/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
104
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Belite Study Site - Westmead
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Recruitment hospital [2]
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Belite Study Site - East Melbourne
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Recruitment hospital [3]
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Belite Study Site - South Brisbane
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Recruitment postcode(s) [1]
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- Westmead
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Recruitment postcode(s) [2]
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- East Melbourne
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Recruitment postcode(s) [3]
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- South Brisbane
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Minnesota
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United States of America
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State/province [3]
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Utah
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Country [4]
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Belgium
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State/province [4]
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Gent
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Country [5]
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China
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State/province [5]
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Beijing
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Country [6]
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China
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State/province [6]
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Shanghai
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Country [7]
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France
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State/province [7]
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Paris
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Country [8]
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Germany
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State/province [8]
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Bonn
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Country [9]
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Germany
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State/province [9]
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Tübingen
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Country [10]
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Hong Kong
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State/province [10]
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Kowloon
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Country [11]
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Netherlands
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State/province [11]
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Nijmegen
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Country [12]
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Switzerland
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State/province [12]
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Basel
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Country [13]
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Taiwan
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State/province [13]
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Taipei
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Country [14]
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Taiwan
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State/province [14]
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Taoyuan City
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Country [15]
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United Kingdom
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State/province [15]
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London
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Country [16]
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United Kingdom
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State/province [16]
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Belite Bio, Inc
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05244304
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05244304
Download to PDF