- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05266014
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
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.
Study Overview
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: tinlarebant
Daily, oral administration of one 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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
From baseline to 24 months
|
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.
|
Up to 24 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Change in atrophic lesion size.
Time Frame: From baseline to 24 months.
|
From baseline to 24 months.
|
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.
Time Frame: Up to 24 months
|
Up to 24 months
|
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.
Time Frame: Up to 24 months
|
Up to 24 months
|
Half-life (t1/2) of tinlarebant in plasma.
Time Frame: Up to 24 months
|
Up to 24 months
|
Time to minimal plasma RBP4 level (Tmin)
Time Frame: Up to 24 months
|
Up to 24 months
|
Minimum concentration of RBP4 (Cmin)
Time Frame: Up to 24 months
|
Up to 24 months
|
Collaborators and Investigators
Sponsor
Collaborators
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- LBS-008-CT02
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Stargazer Pharmaceuticals, Inc.Completed
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Clinical Trials on tinlarebant
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RBP4 Pty LtdBelite Bio, IncCompletedHealthy Volunteer | Dry Age-related Macular DegenerationAustralia