Stargardt Disease: Phase 2 Clinical Trial Enrolling Participants 12 years and older in the USA

We are currently enrolling patients who are 12 years of age and older and who have been diagnosed with Stargardt disease, to participate in a midstage (Phase 2) clinical trial taking place at multiple clinical sites in the United States. If you are interested or know someone who could be interested in participating, please email us at or register your name on our Stargardt registry mailing list.

For the most up to date information about this study, including the list of clinical sites, click on the following link:

About Stargardt Disease and ALK-001

Stargardt disease (STGD1) is a currently untreatable genetic disorder and a leading cause of juvenile blindness affecting approximately 30,000 people in the USA (1 person in 10,000). In its early stage, symptoms include loss of central vision that is uncorrectable with glasses. The condition then progresses to legal blindness (20/200) in the majority of the cases. Most patients experience their first symptoms between the ages of 6 to 30 years of age.

ALK-001 is once-a-day oral drug candidate currently being tested for the treatment of Stargardt disease. In the eyes of patients with Stargardt, toxic vitamin A aggregates called "dimers" form due to a genetic defect. Dimers and their degradation products have been shown to be toxic to the retina and lead to vision loss early in life. Dimers form when two molecules of vitamin A are chemically combined. ALK-001 is vitamin A which has been chemically modified to prevent its aggregation into dimers.

In a Phase 1 clinical trial, we have assessed the safety and pharmacokinetics of ALK-001 in healthy volunteers. (Click here to read details about the Phase 1)


If you are interested in learning more about future clinical trials, please send us an email at Alkeus Pharma intends to run clinical trials in serious diseases such as the following:

Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD)

Dry-AMD is the leading cause of blindness for people over 50. There is no treatment or cure for dry-AMD. The late form of dry-AMD, also called Geographic Atrophy (GA) affects approximately 1 million people in the USA alone. Patients with GA have seriously impaired vision, with decreased visual acuity that is uncorrectable with glasses or lenses. GA progresses with time leading to legal blindness. Peripheral vision is usually preserved.

Intermediate dry-AMD

The intermediate form of dry-AMD affects approximately 8 million patients in the US alone. The severity of the disease is measured by a retinal specialist who attributes a disease grade (from 1 to 4).

If grade 1 and grade 2 have a relatively little chance of progression to late-AMD (dry or wet forms), grade 3 and grade 4 have respectivaly about 25% and 50% chance of progression to late-AMD after 5 years. These two groups of patients are at high risk of complications resulting of late-AMD.

Autosomal Recessive Retinitis Pigmentosa (RP)

Retinitis pigmentosa (RP) forms a group of rare diseases that lead to blindness preceded by night blindness and tunnel vision for several years or decades. The disease affects approximately 100,000 people in the US.
Although multiple genes when mutated can cause Retinitis Pigmentosa, about 5% of the patients with RP have mutated ABCA4 genes, which leads to improper processing of vitamin A by the eye. These patients could potentially benefit from Alkeus Pharma's technology.

Autosomal Recessive Cone-Rod Dystrophy (CORD3)

Cone-Rod dystrophy is a rare disease which affects the cones more seriously than the rods. The disease is usually diagnosed by evaluating the amplitudes of the retina's response to light signals and confirmed genetically. Multiple genes can cause Cone-Rod dystrophies. When the ABCA4 is defective, the disease is thought to be caused by the accelerated formation of toxic vitamin A aggregates in the eye. Because ALK-001 prevents the formation of toxic aggregates in the eye, it could possibly benefit patients with Cone-Rod dystrophy.