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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05266014
Registration number
NCT05266014
Ethics application status
Date submitted
26/01/2022
Date registered
4/03/2022
Titles & IDs
Public title
This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease
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Scientific title
Phase 1/2, Open-Label, Dose-Finding Followed by 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease
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Secondary ID [1]
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LBS-008-CT02
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Stargardt Disease
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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
Experimental: tinlarebant - Daily, oral administration of one tinlarebant.
Treatment: Drugs: tinlarebant
Phase 1b Portion: tinlarebant will be self-administered orally once daily for 2 cycles, 14 days per cycle.
Phase 2 portion: tinlarebant will be self-administered orally once daily for 24 months.
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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 evaluate systemic and ocular safety and tolerability of tinlarebant.
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Assessment method [1]
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To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
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Timepoint [1]
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From baseline to 24 months
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Primary outcome [2]
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The optimal dose for Phase 2.
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Assessment method [2]
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To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.
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Timepoint [2]
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Up to 24 months
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Secondary outcome [1]
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Change in atrophic lesion size.
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Assessment method [1]
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Timepoint [1]
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From baseline to 24 months.
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Secondary outcome [2]
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Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.
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Assessment method [2]
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Timepoint [2]
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Up to 24 months
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Secondary outcome [3]
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Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.
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Assessment method [3]
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Timepoint [3]
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Up to 24 months
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Secondary outcome [4]
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Half-life (t1/2) of tinlarebant in plasma.
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Assessment method [4]
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Timepoint [4]
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Up to 24 months
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Secondary outcome [5]
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Time to minimal plasma RBP4 level (Tmin)
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Assessment method [5]
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Timepoint [5]
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Up to 24 months
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Secondary outcome [6]
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Minimum concentration of RBP4 (Cmin)
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Assessment method [6]
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Timepoint [6]
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Up to 24 months
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Eligibility
Key inclusion criteria
Major
Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.
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Minimum age
12
Years
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Maximum age
18
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 disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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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
12/03/2021
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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
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Actual
15/08/2023
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Sample size
Target
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Accrual to date
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Final
13
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Recruitment in Australia
Recruitment state(s)
NSW,WA
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Recruitment hospital [1]
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Sydney Children's Hospitals Network - Westmead
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Recruitment hospital [2]
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Lions Eye Institute - Perth
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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6009 - Perth
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Recruitment outside Australia
Country [1]
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Taiwan
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State/province [1]
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Taipei
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
RBP4 Pty Ltd
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Address
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Country
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Other collaborator category [1]
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Commercial sector/industry
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Name [1]
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Belite Bio, Inc
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis. This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.
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Trial website
https://clinicaltrials.gov/study/NCT05266014
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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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Country
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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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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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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/NCT05266014