What side effects are associated with this type of intervention?

The most common treatments for Stargardt Disease include vitamin A restriction, gene therapy, and emerging treatments like RBP4 inhibitors such as Tinlarebant. Known side effects of these treatments can vary. For example, vitamin A restriction may lead to deficiencies affecting vision and overall health. Gene therapy can have side effects such as immune reactions and inflammation. Specific side effects of RBP4 inhibitors like Tinlarebant are still under investigation, but potential concerns include effects on vitamin A metabolism and related visual and systemic impacts.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Stargardt Disease. Tinlarebant, an RBP4 inhibitor, is currently being studied for its efficacy in slowing the progression of this condition. Other investigational treatments for Stargardt Disease include gene therapy and stem cell therapy, but these are still in clinical trial phases and have not yet received FDA approval.

What other types of research exist?

Promising novel alternatives to Tinlarebant for Stargardt Disease patients include visual cycle inhibitors like fenretinide and potential treatments involving lutein and zeaxanthin supplements. Additionally, research into the use of potent statins and the drug levodopa, which has shown epidemiological evidence of reducing AMD incidence, may also offer new avenues for treatment.

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Tinlarebant for Stargardt Disease (DRAGON Trial)

Phase 3

Waitlist Available

Research Sponsored by Belite Bio, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

Any prior gene therapy

History of ocular surgery in the study eye in the last 3 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline thru month 24

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called tinlarebant to see if it can slow down eye damage in teenagers with a condition called STGD1. About 90 participants will take tinlarebant for an extended period. The goal is to see if tinlarebant can help prevent their vision from getting worse.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for Tinlarebant are not listed here, common side effects for treatments like these may include irritation at the site of administration, potential allergic reactions, headaches, nausea, or visual disturbances.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have Stargardt disease with a confirmed ABCA4 gene mutation.

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

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I have Stargardt disease with a confirmed ABCA4 gene mutation.

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I am between 12 and 20 years old.

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I am between 12 and 18 years old.

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My vision in the study eye is 20/200 or better and I have specific lesion sizes.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have received gene therapy before.

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I have had eye surgery in the last 3 months.

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I haven't taken any experimental drugs recently.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline thru month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline thru month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline thru month 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

To assess the systemic and ocular safety and tolerability of tinlarebant

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: TinlarebantExperimental Treatment1 Intervention

5 mg tablet taken orally once a day

Group II: PlaceboPlacebo Group1 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.

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Tinlarebant, an RBP4 inhibitor, works by reducing the levels of retinol-binding protein 4 (RBP4), which in turn decreases the delivery of retinol to the retina. This is significant for Stargardt Disease patients because the accumulation of toxic vitamin A derivatives, such as A2E, in the retinal pigment epithelium is a key factor in the progression of the disease. By limiting the supply of retinol, RBP4 inhibitors can potentially slow down the formation of these toxic byproducts, thereby slowing the progression of retinal degeneration and preserving vision.

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