Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623001328662
Ethics application status
Approved
Date submitted
30/11/2023
Date registered
19/12/2023
Date last updated
19/12/2023
Date data sharing statement initially provided
19/12/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
An Open-Label, Single-Arm, 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease
Scientific title
An Open-Label, Single-Arm, 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Subjects with Stargardt Disease
Secondary ID [1] 311070 0
LBS-008-CT08
Universal Trial Number (UTN)
Trial acronym
Linked study record
This record is a extensiton study for ACTRN12621000549820 (LBS-008-CT02).

Health condition
Health condition(s) or problem(s) studied:
Stargardt Disease 332214 0
Condition category
Condition code
Human Genetics and Inherited Disorders 328926 328926 0 0
Other human genetics and inherited disorders
Eye 328927 328927 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Oral Tinlarebant tablet (5mg) once daily for 24 months. Subjects will record the administation of the study drug via daily patient diary.
Intervention code [1] 327518 0
Treatment: Drugs
Comparator / control treatment
NA
Control group
Uncontrolled

Outcomes
Primary outcome [1] 336728 0
To evaluate long-term ocular safety and tolerability of Tinlarebant
Timepoint [1] 336728 0
The outcome will be assessed every 6 months from baseline to 24 months.
Primary outcome [2] 336850 0
To evaluate long-term systemic safety and tolerability of Tinlarebant
Timepoint [2] 336850 0
The outcome will be assessed every 6 months from baseline to 24 months.
Secondary outcome [1] 429485 0
To measure the change in atrophic lesion size
Timepoint [1] 429485 0
The outcome will be assessed every 6 months from baseline to 24 months.

Eligibility
Key inclusion criteria
Subject has completed treatment in LBS-008-CT02 trial and has also completed the tests and assessments required end-of-treatment visit.
Minimum age
12 Years
Maximum age
20 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Ocular surgery in the study eye in the previous 3 months.
2. Use of prescription medications such as Isotretinoin (13-cis-retinoic acid) or other retinol modulators or derivatives
3. Use of any known drugs or supplements that are moderate or strong inhibitors/inducers of cytochrome P450 (CYP) enzymes

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



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

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
14/11/2023
Date of last participant enrolment
Anticipated
31/01/2024
Actual
Date of last data collection
Anticipated
28/02/2026
Actual
Sample size
Target
12
Accrual to date
4
Final
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment outside Australia
Country [1] 26002 0
Taiwan, Province Of China
State/province [1] 26002 0

Funding & Sponsors
Funding source category [1] 315327 0
Commercial sector/Industry
Name [1] 315327 0
RBP4 Pty Ltd
Country [1] 315327 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
RBP4 Pty Ltd
Address
Level 7, 330 Collins Street Melbourne VIC 3000
Country
Australia
Secondary sponsor category [1] 317384 0
None
Name [1] 317384 0
Address [1] 317384 0
Country [1] 317384 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 314249 0
The Sydney Children’s Hospitals Network Human Research Ethics Committee (SCHN HREC)
Ethics committee address [1] 314249 0
Corner Hawkesbury Road and Hainsworth Street, Westmead 2145
Ethics committee country [1] 314249 0
Australia
Date submitted for ethics approval [1] 314249 0
Approval date [1] 314249 0
06/09/2023
Ethics approval number [1] 314249 0
Ethics committee name [2] 314250 0
Bellberry Human Research Ethics Committee
Ethics committee address [2] 314250 0
123 Glen Osmond Road Eastwood SA 5063
Ethics committee country [2] 314250 0
Australia
Date submitted for ethics approval [2] 314250 0
Approval date [2] 314250 0
23/11/2023
Ethics approval number [2] 314250 0

Summary
Brief summary
Since Tinlarebant has not yet been approved, the purpose of this study is to continuously provide Tinlarebant to patients who have completed the 24 months of treatment with Tinlarebant in the previous study (i.e., LBS-008-CT02) with no safety issues.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 130930 0
Prof Fred Chen
Address 130930 0
Lions Eye Institute (2 Verdun Street, Nedlands WA 6009)
Country 130930 0
Australia
Phone 130930 0
+610893810790
Fax 130930 0
Email 130930 0
fred.chen@lei.org.au
Contact person for public queries
Name 130931 0
Prof Fred Chen
Address 130931 0
Lions Eye Institute (2 Verdun Street, Nedlands WA 6009)
Country 130931 0
Australia
Phone 130931 0
+610893810790
Fax 130931 0
Email 130931 0
fred.chen@lei.org.au
Contact person for scientific queries
Name 130932 0
Prof Fred Chen
Address 130932 0
Lions Eye Institute (2 Verdun Street, Nedlands WA 6009)
Country 130932 0
Australia
Phone 130932 0
+610893810790
Fax 130932 0
Email 130932 0
fred.chen@lei.org.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
At this time there is no plan to submit IPD, however, should this change, this record will be updated accordingly


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.