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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
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
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
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
Trial website
https://clinicaltrials.gov/study/NCT05244304
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05244304