Tinlarebant for Stargardt Disease

(DRAGON Trial)

No longer recruiting at 52 trial locations

Overseen By Belitebio - Clinical Trial

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Belite Bio, Inc

Disqualifiers: Ocular disease, Ocular surgery, Gene therapy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called tinlarebant to determine if it can slow the damage caused by Stargardt disease, a genetic eye condition that leads to vision loss. Participants will receive either the tinlarebant pill or a lookalike placebo (a pill with no active medicine) to compare results. The trial seeks young people aged 12 to 20 diagnosed with Stargardt disease and specific eye changes linked to the condition. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you have used any investigational drugs in the last 3 months, you may not be eligible to participate.

Is there any evidence suggesting that tinlarebant is likely to be safe for humans?

Research has shown that tinlarebant is well-tolerated. One study found that using tinlarebant for 24 months was safe, with no major safety issues. Another report highlighted its safety and tolerability, emphasizing its role in reducing harmful substances linked to eye disease. People taking tinlarebant generally did not experience serious side effects. These findings suggest that tinlarebant could be a safe option for treating Stargardt disease. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Stargardt Disease?

Tinlarebant is unique because it targets Stargardt disease differently from current treatments, which mostly focus on managing symptoms rather than addressing the root cause. Unlike the standard care options, which may include vitamin A supplementation or low vision aids, tinlarebant works by potentially slowing the progression of the disease at a cellular level. Researchers are excited about tinlarebant because it is designed to inhibit the accumulation of toxic vitamin A byproducts in the retina, offering hope for a more effective way to preserve vision in people with Stargardt disease.

What evidence suggests that tinlarebant might be an effective treatment for Stargardt disease?

Research has shown that tinlarebant, which participants in this trial may receive, might help treat Stargardt Disease by reducing harmful substances in the eye linked to vitamin A, known as bisretinoids. In studies, tinlarebant consistently lowered a protein called RBP4 by 80-90%, which helps decrease these harmful substances. This reduction can be reversed, allowing for treatment adjustments as needed. Early results from various trials suggest that tinlarebant may slow the growth of damaged areas in the eyes of people with Stargardt Disease. This encouraging evidence supports further research into tinlarebant's effectiveness for this condition. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

Adolescents aged 12-20 with Stargardt Disease (STGD1) and a specific gene mutation. They must have certain eye health criteria, like a lesion size within limits and vision of 20/200 or better. Participants need to agree to follow the study rules and provide informed consent.

Inclusion Criteria

I have Stargardt disease with a confirmed ABCA4 gene mutation.

Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score

My eye condition has been confirmed with a specific imaging test.

Exclusion Criteria

Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L)

I have no eye conditions other than Stargardt disease that could affect treatment assessment.

I have received gene therapy before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tinlarebant 5 mg or placebo orally once a day

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Tinlarebant

Trial Overview

The trial is testing Tinlarebant's effectiveness in slowing down the growth of lesions in the eyes caused by STGD1 compared to a placebo. The main goal is to see if this drug can help preserve vision in affected adolescents.

How Is the Trial Designed?

Group I: Tinlarebant Experimental Treatment 1 Intervention

5 mg tablet taken orally once a day

Group II: Placebo Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

Belite Bio, Inc

Lead Sponsor

Trials

7

Recruited

730+

Published Research Related to This Trial

Stargardt disease is a common macular dystrophy that typically begins in childhood or early adolescence, leading to progressive vision loss, with a prevalence of about 1 in 8000 to 10,000 individuals.

Characteristic features of Stargardt disease include a bull's eye pattern on the fundus and specific changes in autofluorescence imaging, with peripapillary sparing observed in most cases, suggesting a potential resilience in that area against the effects of the ABCA4 gene mutation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stargardt Disease. Tsang, SH., Sharma, T.[2019]

The incidence of Stargardt disease (STGD1) was found to be between 1.67 and 1.95 cases per million per year, with a point prevalence of approximately 1 in 22,000 people in 2018, indicating it is a relatively rare condition.

Late-onset STGD1 (diagnosed at age 45 or older) has increased in recent years, making up 33% of diagnoses from 2014-2018, likely due to improved awareness, but misdiagnosis as age-related macular degeneration was common in this group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stargardt disease: monitoring incidence and diagnostic trends in the Netherlands using a nationwide disease registry. Runhart, EH., Dhooge, P., Meester-Smoor, M., et al.[2022]

Stargardt disease, caused by mutations in the ABCA4 gene, currently lacks effective treatments that have successfully completed clinical trials.

Research is ongoing in various areas, including drugs to reduce harmful vitamin A dimers, stem cell therapies for retinal regeneration, and gene therapy aimed at delivering a functional ABCA4 gene directly into the eye.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[New possibilities in the treatment of Stargardt disease]. Zhorzholadze, NV., Sheremet, NL., Tanas, AS., et al.[2020]

In a study of 42 patients with childhood-onset Stargardt disease (STGD), it was found that this condition leads to severe visual loss and early retinal changes, with 90% of patients carrying at least one disease-causing variant in the ABCA4 gene.

Patients with childhood-onset STGD were more likely to have two harmful variants in the ABCA4 gene compared to those with adult-onset STGD, suggesting that earlier onset may be linked to more severe genetic mutations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and molecular characteristics of childhood-onset Stargardt disease. Fujinami, K., Zernant, J., Chana, RK., et al.[2022]

Stargardt disease (STGD) is caused by mutations in the ABCR gene, which is crucial for retinal function and is expressed primarily in rod photoreceptors.

The study identified 19 different mutations in the ABCR gene among STGD families, confirming its role as the causal gene for this autosomal recessive retinal disorder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy. Allikmets, R., Singh, N., Sun, H., et al.[2022]

Citations

1.

investors.belitebio.com

investors.belitebio.com/news-releases/news-release-details/belite-bio-announces-completion-dragon-2-year-phase-3-trial-oral

Belite Bio Announces Completion of DRAGON, a 2-Year ...

Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06388083

NCT06388083 | A Phase 2/3 Study to Evaluate the Efficacy ...

See Delay Results Type in the Results Data ... A Phase 2/​3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (DRAGON II).

3.

ophthalmologytimes.com

ophthalmologytimes.com/view/belite-bio-concludes-phase-3-dragon-study-for-stargardt-disease

Belite Bio concludes phase 3 DRAGON study for Stargardt ...

Belite Bio completes its phase 3 trial for tinlarebant, a potential first treatment for Stargardt disease, with results expected in late 2025.

4.

iovs.arvojournals.org

iovs.arvojournals.org/article.aspx?articleid=2797066

Safety, Tolerability, and Efficacy of Tinlarebant from the 24 ...

Results : Tinlarebant produced a sustained reduction of RBP4 (80-90%) throughout the treatment period which was reversible during 28 days of drug cessation.

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05244304

Study Details | NCT05244304 | Phase 3, Randomized, ...

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 ...

6.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/belite-bio-stargardt-disease/

Belite Bio wraps up Phase III trial of Stargardt disease ...

In July 2025, Belite Bio completed the enrolment for its 24-month PHOENIX Phase III trial, assessing the tolerability and safety of Tinlarebant ...