Overview

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

Status:
Completed

Trial end date:
2016-12-13

Target enrollment:

Participant gender:

Summary

This was a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who were naïve to any form of cysteamine treatment. Participants received RP103 treatment for at least 12 months. U.S. participants transitioned to the commercially approved drug PROCYSBI®. In Brazil, after at least 12 months of study participation and upon approval by the Brazilian regulatory authorities, participants were eligible to transition to a post-study drug supply program, and continue to receive the drug at no personal cost.

Phase:

Phase 3

Details

Lead Sponsor:

Horizon Pharma USA, Inc.
Raptor Pharmaceuticals Inc.

Treatments:

Cysteamine