Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1. 1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2. 2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3. 3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4. 4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5. 5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6. 6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.