Tinlarebant for Stargardt Disease

(DRAGON II Trial)

Recruiting at 16 trial locations

Overseen By Belite Bio Clinical Operations

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Belite Bio, Inc

Disqualifiers: Ocular disease, Ocular surgery, Gene therapy

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

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called tinlarebant for Stargardt Disease, a genetic eye condition that causes vision loss. The main goal is to determine if tinlarebant is safe and effective. Participants will take either tinlarebant or a placebo (a pill with no active medicine) to compare results. This trial suits individuals diagnosed with Stargardt Disease who have a specific gene mutation linked to it and experience vision problems due to lesions in their eyes. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to a potential breakthrough in treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research has shown that tinlarebant is generally safe for people. In a previous 24-month study, tinlarebant proved to be safe and easy for participants to handle. They experienced a steady and reversible drop in a protein linked to vitamin A toxins, which cause issues in Stargardt Disease. Another study with teenagers demonstrated that tinlarebant was safe over 15 months. These findings suggest that tinlarebant has a good safety record and is well-tolerated. ^{1 2 3 4 5}

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

Researchers are excited about tinlarebant for Stargardt disease because it offers a novel approach compared to the current standard treatments like vitamin A restriction and antioxidant supplements. Tinlarebant works by inhibiting the formation of toxic vitamin A derivatives that accumulate in the retina, which is different from other options that mainly focus on managing symptoms rather than the underlying cause. This innovative mechanism has the potential to slow down the progression of vision loss, giving hope for a more effective management strategy for those affected by this genetic eye condition.

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, helps reduce the build-up of harmful substances related to vitamin A, known as bisretinoids, believed to damage the eyes in Stargardt Disease. In an earlier study, patients taking tinlarebant experienced an 80-90% decrease in a protein associated with these harmful substances, and this effect persisted throughout the treatment. This suggests that tinlarebant might help slow Stargardt Disease by addressing one of its main causes. While more research is needed, these findings offer promising early evidence of tinlarebant's potential effectiveness. ^{2 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Stargardt Disease, a type of inherited eye disorder that causes vision loss. Participants should have a diagnosis of this condition to be eligible.

Inclusion Criteria

With your glasses or contacts, can you still read some of the big letters on an eye-chart (not totally blind in either eye)?

Have you avoided any eye surgery or shots in either eye during the past three months?

Exclusion Criteria

Are you taking high-dose vitamin A, Accutane®, Soriatane®, or similar retinoid medicines?

Besides Stargardt, have you ever been treated for glaucoma, diabetic eye disease, macular degeneration, or other serious eye problems?

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1b Treatment

Open-label evaluation of pharmacokinetics, pharmacodynamics, safety, and tolerability of daily doses of 5 mg tinlarebant for 7 days in Japanese subjects

1 week

Phase 2/3 Treatment

Randomized, double-masked, placebo-controlled evaluation of safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant for 24 months

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tinlarebant

Trial Overview The study is testing the effectiveness and safety of a drug called Tinlarebant compared to a placebo in treating Stargardt Disease. It's designed to see if Tinlarebant can help with symptoms or slow down progression.

How Is the Trial Designed?

Group I: LBS-008, 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 prepared similarly

Who Is Running the Clinical Trial?

Belite Bio, Inc

Lead Sponsor

Trials

7

Recruited

730+

Published Research Related to This Trial

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]

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]

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]

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]

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/NCT05244304

Study Details | NCT05244304 | Phase 3, Randomized, ... Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up. Official Title. Phase 3, ...

3. 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.

4. 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.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06388083

NCT06388083 | A Phase 2/3 Study to Evaluate the Efficacy ... The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease.

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 ...

7. retinalphysician.com retinalphysician.com/issues/2025/october/novel-therapies-for-stargardt-disease/

Novel Therapies for Stargardt Disease In a phase 1b/2 trial, tinlarebant demonstrated a favorable safety and tolerability profile in adolescents (ages 12-18) with STGD. At 15 months, ...