STGD1Phase 2/3DRAGON IINCT06388083
A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease
A Phase 2/3 study testing Tinlarebant in people with STGD1 and Stargardt Disease 1, run by Belite Bio, Inc at 18 sites in 3 countries.
Trial summary
The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease Read more about this study
- Who is it for?
- Ages 12 to 20 years
- Conditions the study is for
- STGD1, 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
- 60 participants
- Where
- 18 sitesin 3 countries
- Study dates
- 31 Jul 2024to Feb 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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
- Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
- Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
- Minimum BCVA is required in the study eye
Who cannot join
- Any ocular disease other than STGD1 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.
- Any prior gene therapy.
Trial locations and contacts
18 sites in 3 countries.
18 sites in 3 countries
United States14 sites
Belite Study Site - US13
Austin, Texas, 78705
Belite Study Site - US01
Boston, Massachusetts, 02115
Belite Study Site - US03
Dallas, Texas, 75231
Belite Study Site - US02
Durham, North Carolina, 27705
Belite Study Site - US11
Edina, Minnesota, 55435
Belite Study Site - US05
La Jolla, California, 92093
Belite Study Site - US06
Miami, Florida, 33136
Belite Study Site - US14
New York, New York, 10032
Belite Study Site - US07
Philadelphia, Pennsylvania, 19107
Belite Study Site - US08
Phoenix, Arizona, 85020
Belite Study Site - US04
Rochester, Minnesota, 55905
Belite Study Site - US09
San Francisco, California, 94158
Belite Study Site - US12
The Woodlands, Texas, 77384
Belite Study Site - US10
Westbury, New York, 11590
Japan3 sites
Belite Study Site
Kobe
Belite Study Site
Kyoto
Belite Study Site
Tokyo
About this study
Official title: A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease
The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease
Read the detailed description
This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.
- Conditions
- STGD1, 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
Also called LBS-008
5 mg tablet
PlaceboDrug
Placebo tablets
| Study group | Role | What they receive |
|---|---|---|
| LBS-008, Tinlarebant | Experimental | 5 mg tablet taken orally once a day Receives: Drug: Tinlarebant |
| Placebo | Placebo comparator | Placebo tablets for tinlarebant 5 mg prepared similarly Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
Measured: From baseline to Month 24
Secondary outcomes (2)
To measure the annualized rate of change in total area of atrophy
Measured: From baseline to Month 24
Change in BCVA measured by the ETDRS method
Measured: From baseline to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2024
- Study start31 Jul 2024
- Primary completionFeb 2028 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for STGD1Studies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Belite Bio, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06388083
- Sponsor's protocol ID
- LBS-008-CT07
- Acronym
- DRAGON II
- Status last verified
- Mar 2026
- First submitted
- 23 Apr 2024
- Last updated
- 12 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease
- ClinicalTrials.gov ID
- NCT06388083
- Acronym / sponsor ID
- DRAGON II / LBS-008-CT07
- Conditions
- STGD1, Stargardt Disease 1
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Belite Bio, IncIndustry (pharma, biotech, devices)
- Study start
- 31 Jul 2024 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 29 Apr 2024 (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 goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease
Detailed description
This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LBS-008, Tinlarebant | Experimental | Drug: Tinlarebant5 mg tablet taken orally once a day |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo tablets for tinlarebant 5 mg prepared similarly |
Who may qualify
- Ages
- 12 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
- Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
- Minimum BCVA is required in the study eye
Key exclusion criteria (3 listed)
- Any ocular disease other than STGD1 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.
- Any prior gene therapy.
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 |
|---|---|---|
| TinlarebantAlso: LBS-0085 mg tablet | Drug | LBS-008, Tinlarebant |
| PlaceboPlacebo tablets | Drug | Placebo |
Primary outcome
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy
Time frame: From baseline to Month 24
2 secondary outcomes
To measure the annualized rate of change in total area of atrophy
Time frame: From baseline to Month 24
Change in BCVA measured by the ETDRS method
Time frame: From baseline to Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2026.
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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.
18 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Tinlarebant in people with STGD1 and Stargardt Disease 1, run by Belite Bio, Inc at 18 sites in 3 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 3 inclusion and 3 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: under way, not looking for participants, as of the sponsor's last update on 12 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Japan, United Kingdom. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
This study is still under way. Results are not available in the source record yet.
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 the annualized rate of change from baseline lesion size in aggregate area of atrophy (time frame: From baseline to 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 12 Mar 2026.