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Your journey
1What's a trial
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3Review
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More info
You can access this
clinical trial
if you have
Cystinosis
and you are
under 6
years old
3
This is a trial in the final phase before the treatment is released on the market.
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The purpose

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.

Provided treatments

  • Drug: RP103

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01744782. The sponsor of the trial is Horizon Pharma USA, Inc. and it is looking for 17 volunteers for the current phase.
Official trial title:
An Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With Cystinosis