Stargardt Disease 1Phase 3DRAGONNCT05244304
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
A Phase 3 study testing Tinlarebant in people with Stargardt Disease 1, run by Belite Bio, Inc at 19 sites in 11 countries.
Trial summary
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1 Read more about this study
- Who is it for?
- Ages 12 to 20 years
- Conditions the study is for
- Stargardt Disease 1
- What is being tested?
- Tinlarebant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 104 participants
- Where
- 19 sitesin 11 countries
- Study dates
- 28 Mar 2022to 11 Sep 2025
- Phase
- Phase 3
- 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 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female subjects 12 to 20 years old, inclusive.
- Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
- Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
- Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
- Subject agrees to comply with all protocol requirements.
Who cannot join
- Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect.
- History of ocular surgery in the study eye in the last 3 months.
- Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
- Any prior gene therapy.
- Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).
Trial locations and contacts
19 sites in 11 countries.
19 sites in 11 countries
United States3 sites
Belite Study Site
Minneapolis, Minnesota, 55435
Belite Study Site
Palo Alto, California, 94303
Belite Study Site
Salt Lake City, Utah, 84132
Australia3 sites
Belite Study Site
East Melbourne, Victoria
Belite Study Site
South Brisbane
Belite Study Site
Westmead, New South Wales
United Kingdom2 sites
Belite Study Site
London
Belite Study Site
Southampton
About this study
Official title: Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
Read the detailed description
Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.
- Conditions
- Stargardt Disease 1
- Being tested
- Tinlarebant, Placebo
- 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
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.
PlaceboDrug
Not active drug
| Study group | Role | What they receive |
|---|---|---|
| Tinlarebant | Experimental | 5 mg tablet taken orally once a day Receives: Drug: Tinlarebant |
| Placebo | Placebo comparator | Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Measured: Baseline thru month 24
Secondary outcomes (6)
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Measured: Baseline thru month 24
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Measured: Baseline thru month 24
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Measured: Baseline thru month 24
To measure change in plasma concentration of RBP4 levels (μM) from baseline
Measured: Baseline thru month 24
The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Measured: Baseline thru month 24
To assess the systemic and ocular safety and tolerability of tinlarebant
Frequency, duration, and severity of AEs
Measured: Baseline thru month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2022
- Study start28 Mar 2022
- Primary completion4 Aug 2025
- Study completion11 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt Disease 1Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 104 participants (actual)
Sponsor and investigators
- Lead sponsor
- Belite Bio, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05244304
- Sponsor's protocol ID
- LBS-008-CT03
- Acronym
- DRAGON
- Status last verified
- Jul 2026
- First submitted
- 20 Jan 2022
- Last updated
- 23 Jul 2026
- 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 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
- ClinicalTrials.gov ID
- NCT05244304
- Acronym / sponsor ID
- DRAGON / LBS-008-CT03
- Conditions
- Stargardt Disease 1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 104 participants (actual)
- Lead sponsor
- Belite Bio, IncIndustry (pharma, biotech, devices)
- Study start
- 28 Mar 2022 (actual)
- Primary completion
- 4 Aug 2025 (actual)
- Study completion
- 11 Sep 2025 (actual)
- First posted
- 17 Feb 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.
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1
Detailed description
Approximately 90 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
| Arm | Type | Receives |
|---|---|---|
| Tinlarebant | Experimental | Drug: Tinlarebant5 mg tablet taken orally once a day |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance. |
Who may qualify
- Ages
- 12 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Male or female subjects 12 to 20 years old, inclusive.
- Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
- Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
- Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
- Subject agrees to comply with all protocol requirements.
Key exclusion criteria (5 listed)
- Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect.
- History of ocular surgery in the study eye in the last 3 months.
- Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
- Any prior gene therapy.
- Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).
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 |
|---|---|---|
| TinlarebantTinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration. | Drug | Tinlarebant |
| PlaceboNot active drug | Drug | Placebo |
Primary outcome
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time frame: Baseline thru month 24
6 secondary outcomes
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time frame: Baseline thru month 24
To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline
Time frame: Baseline thru month 24
To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline
Time frame: Baseline thru month 24
To measure change in plasma concentration of RBP4 levels (μM) from baseline
Time frame: Baseline thru month 24
The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline
Time frame: Baseline thru month 24
To assess the systemic and ocular safety and tolerability of tinlarebant
Time frame: Baseline thru month 24
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 23 Jul 2026.
- Individual participant dataSharing planned: No
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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.
19 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tinlarebant in people with Stargardt Disease 1, run by Belite Bio, Inc at 19 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. 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 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Australia, China, Germany, Taiwan and 6 more countries. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline (time frame: Baseline thru month 24). 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 23 Jul 2026.