An Open-Label Safety and Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

Official Title: An Open-Label Safety and Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

This is a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who are naïve to any form of cysteamine treatment. Subjects will receive RP103 treatment for at least 12 months and will continue to participate in the study and receive RP103, until it is available through the appropriate marketing approval (In the US, following FDA approval; In Brazil, once it is a regularly registered medication and available at no personal cost), or a subject withdraws or is withdrawn from the study, or the Sponsor terminates development of RP103 for cystinosis. The purpose of this study is to gather information about the safety and effectiveness (how well it works to treat cystinosis) of a new drug called RP103.
NCT01744782
Langman, Craig B., MD
Interventional
No

Contact Information:

Heather Price, MS

773.755.6368