This observational study is looking at how cysteamine (Cystagon; ProCysBi) affects people with cystinosis, an inherited disease that can cause poor growth and kidney problems. Cysteamine is a medication that helps reduce the buildup of a substance called cystine in the body's cells, which is thought to cause the damage in cystinosis. You might be able to join if you have a diagnosis of cystinosis, confirmed by a specific level of cystine in your white blood cells. The study aims to gather knowledge and advice for individuals with cystinosis and the wider community. The current status of this study is unclear, and it plans to include up to 330 participants of all ages, from 1 week to 115 years old.

Study design

This is an observational study, meaning researchers are watching how cysteamine affects participants over time. It plans to enroll up to 330 participants.

What's involved

If you participate, you would be admitted to the NIH Clinical Center every two years for 1-3 days. During these visits, a series of tests will be performed to monitor the effectiveness of cysteamine.

Compensation

Not stated in the trial record.

Follow-up

Participants are followed up every two years, with the primary endpoint measured at these follow-up intervals.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.