Tinlarebant for Stargardt Disease

(DRAGON II Trial)

Recruiting at 3 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

Trial Summary

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.

How is the drug Tinlarebant unique for treating Stargardt disease?

Tinlarebant is unique because it aims to reduce the accumulation of vitamin A dimers and lipofuscin, which are substances that build up in the eyes of people with Stargardt disease, potentially slowing the progression of vision loss. Currently, there are no standard treatments that have fully passed clinical trials for this condition. ^{1 2 3 4 5}

What is the purpose of this trial?

The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

Eligibility Criteria

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

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

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

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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, 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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Stargardt Disease. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Stargardt disease: monitoring incidence and diagnostic trends in the Netherlands using a nationwide disease registry. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Clinical and molecular characteristics of childhood-onset Stargardt disease. [2022]

4. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[New possibilities in the treatment of Stargardt disease]. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy. [2022]