Trial of Cysteamine in Treatment of Corneal Cystine Accumulation in Cystinosis

The Emmes Company, Llc, Rockville MD

Investigators

Abstract

Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis This study will continue to evaluate the long term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis. These drops are not sold commercially and are available only through this study. New patients may enroll in the study to obtain them. Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks.