Stargardt DiseasePhase 1/2NCT05266014
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
A Phase 1/2 study testing tinlarebant in people with Stargardt Disease, run by RBP4 Pty Ltd at 3 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 18 years
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- tinlarebant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 12 Mar 2021to 15 Aug 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Major Inclusion Criteria:
Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.
Major 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.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Australia2 sites
Lions Eye Institute
Perth, Western Australia, 6009
Sydney Children's Hospitals Network
Westmead, New South Wales, 2145
About this study
Official 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
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.
- Conditions
- Stargardt Disease
- Being tested
- tinlarebant
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tinlarebantDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| tinlarebant | Experimental | Daily, oral administration of one tinlarebant. Receives: Drug: tinlarebant |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate systemic and ocular safety and tolerability of tinlarebant.
To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
Measured: From baseline to 24 months
The optimal dose for Phase 2.
To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.
Measured: Up to 24 months
Secondary outcomes (6)
Change in atrophic lesion size.
Measured: From baseline to 24 months.
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.
Measured: Up to 24 months
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.
Measured: Up to 24 months
Half-life (t1/2) of tinlarebant in plasma.
Measured: Up to 24 months
Time to minimal plasma RBP4 level (Tmin)
Measured: Up to 24 months
Minimum concentration of RBP4 (Cmin)
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2022
- Study start12 Mar 2021
- Primary completion15 Aug 2023
- Study completion15 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- RBP4 Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Belite Bio, Inc
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05266014
- Sponsor's protocol ID
- LBS-008-CT02
- Status last verified
- Apr 2024
- First submitted
- 26 Jan 2022
- Last updated
- 12 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05266014
- Acronym / sponsor ID
- LBS-008-CT02
- Conditions
- Stargardt Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- RBP4 Pty LtdIndustry (pharma, biotech, devices)
- Collaborators
- Belite Bio, Inc
- Study start
- 12 Mar 2021 (actual)
- Primary completion
- 15 Aug 2023 (actual)
- Study completion
- 15 Aug 2023 (actual)
- First posted
- 4 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tinlarebant | Experimental | Drug: tinlarebantDaily, oral administration of one tinlarebant. |
Who may qualify
- Ages
- 12 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tinlarebantPhase 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. | Drug | tinlarebant |
Primary outcome
To evaluate systemic and ocular safety and tolerability of tinlarebant.
Time frame: From baseline to 24 months
To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
The optimal dose for Phase 2.
Time frame: Up to 24 months
To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.
6 secondary outcomes
Change in atrophic lesion size.
Time frame: From baseline to 24 months.
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.
Time frame: Up to 24 months
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.
Time frame: Up to 24 months
Half-life (t1/2) of tinlarebant in plasma.
Time frame: Up to 24 months
Time to minimal plasma RBP4 level (Tmin)
Time frame: Up to 24 months
Minimum concentration of RBP4 (Cmin)
Time frame: Up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2024.
- Individual participant dataSharing planned: No
Related trials for Stargardt Disease
RecruitingPhase 1
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Stargardt Disease, Stargardt Macular Dystrophy
Active, not recruitingPhase 1/2
Oral Metformin for Treatment of ABCA4 Retinopathy
ABCA4 Retinopathy, Stargardt Disease
RecruitingObservational study
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults
Stargardt Disease, Stargardt Macular Dystrophy
RecruitingObservational study
Rod and Cone Mediated Function in Retinal Disease
Retinal Degeneration, Retinitis Pigmentosa
RecruitingObservational study
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)
Stargardt Disease, Stargardt Disease 1
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing tinlarebant in people with Stargardt Disease, run by RBP4 Pty Ltd at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Australia, Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate systemic and ocular safety and tolerability of tinlarebant. (time frame: From baseline to 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2024.