Stargardt Disease Clinical Trials 2023

What Are Stargardt-Disease Clinical Trials

Stargardt disease is a genetic condition that causes fatty material buildup on the macula, the retina’s central area, creating vision loss. Central vision is the first area to typically be impacted, and many patients maintain their peripherical vision. However, there are rare cases where total vision loss occurs.

Stargardt-disease symptoms include:

• Central vision loss

• Light sensitivity

• Lengthy periods for eyes to adjust to light changes

• Hazy or black spots in the central vision

• Color blindness

Stargardt disease is inherited. Symptoms typically emerge in childhood, but some are not impacted until adulthood.

In 1997, FFB-funded researchers identified changes in the gene ABCA4 as primarily responsible for the degenerative condition. However, it wasn’t until 2001 that researchers found the condition could also be caused by mutations to ELOVL4.

Why Is Stargardt Disease Being Studied Through Clinical Trials?

There are approximately 30,000 people in the United States with Stargardt disease. According to the NLM (National Library of Medicine), it impacts 1 in 8-10,000 individuals worldwide, making it the most common form of juvenile macular degeneration.

Researchers are examining Stargate disease hoping to find treatments to cure the condition or slow its progression. Various trials are looking into possible:

• Oral medications

• Gene therapies

• Stem cell therapies

• C5 complement inhibitor

What Are The Types Of Treatments Available For Stargardt Disease?

Currently, Stargardt disease has no treatment. However, the National Eye Institute recommends the following to slow down the degeneration:

• Don’t smoke

• Don’t take supplements with extra vitamin A

• Use hats and sunglasses outside to protect your eyes

However, clinical trials are underway in hopes of discovering treatments. For example, Case Western Reserve University researchers have been investigating methods to deliver gene therapy. Thus far, it has only been tested on mice and has yet to progress to clinical trials on human patients.

What Are Some Recent Breakthrough Clinical Trials For Stargardt Disease?

2023: Tinlarebant In Adolescents: Research funded by Belite Bio has conducted Phase 1b/2 student on LBS-008, also known as Tinlarebant. The oral medication has been given an FDA fast-track designation and greenlighted a Phase 3 trial. If successful, patients could take the therapy orally rather than an injection directly into the eye.

2019: ALK-001 – Researchers funded by Alkeus Pharmaceuticals developed a chemically-modified form of vitamin A, ALK-001 (C20-D3-vitamin A), that slows retinal degenerative diseases, including Stargate. The results of Phase 2, a double-masked, randomized, placebo-controlled trial, led to AKL-001 being given Breakthrough Therapy Designation by the FDA. It is now in the Phase 3 stage.

Who Are Some Of The Key Opinion Leaders / Researches In Stargardt-Disease Clinical Trial Research?

Professor Zheng-Rong Lu researches molecular imaging, drug delivery, and nanotechnology at Case Western Reserve University, appointed in the School of Medicine and School of Engineering. He is the lead researcher investigating how chemically modified lipids can deliver gene therapy to treat Stargardt disease.

Professor David Gamm researches degenerative diseases of the retina at the Waisman Center at the University of Wisconsin-Madison. At his laboratory, he looks into using stem cell technology to help reverse or delay effects from conditions such as Stargardt disease.