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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05244304




Registration number
NCT05244304
Ethics application status
Date submitted
20/01/2022
Date registered
17/02/2022

Titles & IDs
Public title
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
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
Secondary ID [1] 0 0
LBS-008-CT03
Universal Trial Number (UTN)
Trial acronym
DRAGON
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Stargardt Disease 1 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Timepoint [1] 0 0
Baseline thru month 24
Secondary outcome [1] 0 0
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Timepoint [1] 0 0
Baseline thru month 24
Secondary outcome [2] 0 0
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Timepoint [2] 0 0
Baseline thru month 24
Secondary outcome [3] 0 0
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Timepoint [3] 0 0
Baseline thru month 24
Secondary outcome [4] 0 0
To measure change in plasma concentration of RBP4 levels (µM) from baseline
Timepoint [4] 0 0
Baseline thru month 24
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
Baseline thru month 24
Secondary outcome [6] 0 0
To assess the systemic and ocular safety and tolerability of tinlarebant
Timepoint [6] 0 0
Baseline thru month 24

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.
Minimum age
12 Years
Maximum age
20 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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).

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2025
Actual
Sample size
Target
Accrual to date
Final
104
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Belite Study Site - Westmead
Recruitment hospital [2] 0 0
Belite Study Site - East Melbourne
Recruitment hospital [3] 0 0
Belite Study Site - South Brisbane
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- East Melbourne
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Utah
Country [4] 0 0
Belgium
State/province [4] 0 0
Gent
Country [5] 0 0
China
State/province [5] 0 0
Beijing
Country [6] 0 0
China
State/province [6] 0 0
Shanghai
Country [7] 0 0
France
State/province [7] 0 0
Paris
Country [8] 0 0
Germany
State/province [8] 0 0
Bonn
Country [9] 0 0
Germany
State/province [9] 0 0
Tübingen
Country [10] 0 0
Hong Kong
State/province [10] 0 0
Kowloon
Country [11] 0 0
Netherlands
State/province [11] 0 0
Nijmegen
Country [12] 0 0
Switzerland
State/province [12] 0 0
Basel
Country [13] 0 0
Taiwan
State/province [13] 0 0
Taipei
Country [14] 0 0
Taiwan
State/province [14] 0 0
Taoyuan City
Country [15] 0 0
United Kingdom
State/province [15] 0 0
London
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Belite Bio, Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05244304