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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/NCT05266014




Registration number
NCT05266014
Ethics application status
Date submitted
26/01/2022
Date registered
4/03/2022
Date last updated
12/04/2024

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
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
Secondary ID [1] 0 0
LBS-008-CT02
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Stargardt Disease 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

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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To evaluate systemic and ocular safety and tolerability of tinlarebant.
Timepoint [1] 0 0
From baseline to 24 months
Primary outcome [2] 0 0
The optimal dose for Phase 2.
Timepoint [2] 0 0
Up to 24 months
Secondary outcome [1] 0 0
Change in atrophic lesion size.
Timepoint [1] 0 0
From baseline to 24 months.
Secondary outcome [2] 0 0
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.
Timepoint [2] 0 0
Up to 24 months
Secondary outcome [3] 0 0
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.
Timepoint [3] 0 0
Up to 24 months
Secondary outcome [4] 0 0
Half-life (t1/2) of tinlarebant in plasma.
Timepoint [4] 0 0
Up to 24 months
Secondary outcome [5] 0 0
Time to minimal plasma RBP4 level (Tmin)
Timepoint [5] 0 0
Up to 24 months
Secondary outcome [6] 0 0
Minimum concentration of RBP4 (Cmin)
Timepoint [6] 0 0
Up to 24 months

Eligibility
Key inclusion criteria
Major

Subject must have clinically diagnosed Stargardt disease with at least one mutation
identified in the ABCA4 gene.

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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/08/2023
Sample size
Target
Accrual to date
Final
13
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
Sydney Children's Hospitals Network - Westmead
Recruitment hospital [2] 0 0
Lions Eye Institute - Perth
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
Taiwan
State/province [1] 0 0
Taipei

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
RBP4 Pty Ltd
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Belite Bio, Inc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05266014
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