This trial is evaluating whether Emixustat will improve 1 primary outcome and 11 secondary outcomes in patients with Stargardt Disease. Measurement will happen over the course of 24 months.
This trial requires 194 total participants across 2 different treatment groups
This trial involves 2 different treatments. Emixustat is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.
"The current research suggests that genetic counseling and the use of an individualized approach can yield benefits for participants while enhancing their life and providing emotional support." - Anonymous Online Contributor
"This autosomal recessive form of inherited retinal disease results in slowly progressive loss of central vision with the development of peripheral visual hallucinations, inappropriate (distorted or enhanced) responses to light and color changes (i.e. the presence and intensity of color hues)." - Anonymous Online Contributor
"Stargardt Disease is a genetic disorder, and the only current treatment is to stop the disease from affecting your retina. There are not many therapies that have been proven to work for Stargardt Disease - but there are a number of devices that have been described, and shown to be effective in small trials. These devices include: the Alpenrod (Optovision Corp), the Laserdisc (LaserVision), the Retimask (IOP Labs), the Gaze-Shutter, the Laser-Shutter, the Stereopix Mini (Alcon), the Eye-Guard Laser (Lerner Labs), and the Laserblitz (Mediflex)." - Anonymous Online Contributor
"There is no single cause for Stargardt disease, but three major genes, ABCA4, CNGB3, and ABCA4R1, have been associated with retinal degeneration. It is important that clinicians understand that more than one gene and environmental condition may alter risk." - Anonymous Online Contributor
"This is the first in-depth analysis of Stargardt disease in the US, examining patient populations and their burden of disease (using ICD-10 diagnostic codes, nationally available health insurance data, and self-reported data). The study illustrates the significant burden of disease for the Stargardt patient population. It also highlights the opportunity to optimize clinical care with disease prevention, by utilizing genetic and disease-modifying therapies at risk of advancing slowly." - Anonymous Online Contributor
"Stargardt disease is an incurable neurodegenerative disease primarily affecting the macular region that can start in children. In a recent study, findings, the patients exhibited characteristic symptoms of strabismus, nystagmus, scotoma and a high incidence of retinal dystrophies." - Anonymous Online Contributor
"Emixustat produced significant improvements in quality of life for patients with Stargardt disease. We anticipate that this effect may translate into a subjective decrease in the burden of visual disability for these patients, with a potential for improved patient acceptance of stargardt disease management and for treatment in trials and real-life situations." - Anonymous Online Contributor
"New discoveries would be helpful for treating the disease, but currently, there is no treatment available. But there are advances in understanding the cause of Stargardt disease, which is that certain metabolites produced by certain cells are toxic to retinal neurons. The research on the cause is making a big headway. One of the new treatments is a treatment for the buildup of toxic metabolites in the bodies. Research has shown that there are some compounds in the eye or in the retina that act as “chemical chaperones”. Compounds that work as a chemical chaperone help to counteract the toxic compounds. A great deal of research into how to use compounds as a chaperone is in order to treat Stargardt disease." - Anonymous Online Contributor
"There are two well-known possible causes to Stargardt disease; a ncoding mutation in the ABCA4 gene and a defect in the transport of vitamin A into retinal pigment epithelial cells. As many as 80% of Stargardt patients have the codon mutation in the ABCA4 gene. Many people have normal BRAIN MAGNIFISATION AND MEGAPACKILOUS BLOOD PRESSURE: INFRACT WITH STRARGARDT DISEASE? answer: The current study indicates that most patients with Stargardt Disease suffer from a secondary end point (brain-associated vascular complications)." - Anonymous Online Contributor
"Results from a recent paper of this review suggest that the use of emixustat in combination with other anti-VEGF drugs and/or other drugs should be studied. For patients with RDS/ Stargardt disease that have been treated with any anti-VEGF drug and/or any other drug, emixustat may be an effective treatment. However, this will need to be studied in a number of larger, randomized, controlled trials." - Anonymous Online Contributor
"The authors concluded that these data suggest that emixustat is an inhibitor of both the ABCA4 and the ABCG2 transporter enzymes, explaining its efficacy in decreasing AUC. The data also suggest that the ABCA4 and ABCG2 transporters are unlikely to the pharmacokinetic or pharmacodynamic targets of EMX for its antiproliferative properties in preclinical models of cancer." - Anonymous Online Contributor
"It is difficult to know how to inform patients, their families and optometrists on the potential value of research that involves a placebo control component. Patients have often not been involved in research, therefore the value of taking part in a placebo-controlled clinical trial is not clear. Further, there is concern about the amount of work for which potential studies may be too expensive. It is difficult for patients to understand the value of research in a disorder they are aware of having; and it is difficult to ensure the right people take part in a study. As there are no long-term outcome studies in Stargardt disease, we are largely relying upon research to create answers about the prognosis of the disorder." - Anonymous Online Contributor