At a glance
ClinicalIndex Comparison Record- ✓Documented clinical diagnosis of cystinosis
- ✓Age 2 years or older
- ✓Able to travel to NIH for baseline exam, 1-year follow-up, and every 2 years thereafter
- ✓Able to cooperate for slit-lamp examination
- ✕Unable to travel to NIH for baseline exam, 1-year follow-up, and biennial follow-up
- ✕Unable to cooperate with slit-lamp examination
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
In Brief
A Phase 2 clinical trial evaluating Cysteamine for Cystinosis. Completed, enrolled 328 participants across 1 site.
Detailed Summary
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. Patients enrolled in a NIH study on cystinosis are receiving the drug cysteamine. Taken by mouth, this drug reduces cystine in some tissues, but not in the cornea. This study began in 1986 to test whether cysteamine eye drops could prevent or reduce corneal cystine crystals in these patients. The drops have been very effective in removing crystals and reducing pain in patients who take the medication as directed. Patients who do not take the medication as prescribed do not benefit. After the effectiveness of the drops was proven, the main purpose was modified to continue to evaluate the long-term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis until the drops are approved by the Food and Drug Administration (FDA). When the New Drug Application (NDA) for the Sigma-Tau standard formulation is granted, this protocol will be terminated.