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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief Summary: 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. US subjects will transition to the commercially approved drug PROCYSBI®. In Brazil, after at least 12 months of study participation and upon approval by the Brazilian regulatory authorities, subjects will be eligible to transition to a post study drug supply program, and continue to receive the drug at no personal cost.

Detailed Summary:

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.

In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of an older, already approved drug called Cystagon® (cysteamine bitartrate) reduces cystine in the body. RP103 has the same active ingredient as Cystagon® and is designed to reduce cystine in a similar way that Cystagon® does. RP103 is also different from Cystagon®: Instead of the cysteamine bitartrate being absorbed from the stomach, RP103 is designed to be absorbed from the small intestine. This may make the effects of the drug last longer, so that it can be taken twice a day instead of four times a day like Cystagon®.

To decide if RP103 is effective, the study will look at two types of blood tests. One test is pharmacodynamics (PD), which measures the amount of white blood cell (WBC) cystine after taking study drug. WBC cystine is a laboratory test used to find out if cysteamine bitartrate is reducing cystine levels in the body. The second test is pharmacokinetics (PK), which measures the amount of cysteamine in the blood after taking the drug.


Sponsor: Horizon Pharma USA, Inc.

Current Primary Outcome: White Blood Cell (WBC) Cystine Levels [ Time Frame: 12 months minimum ]

Steady-state cysteamine-trough WBC cystine levels 30 minutes post RP103 dose at each study visit.


Original Primary Outcome:

  • White Blood Cell (WBC) Cystine Levels [ Time Frame: 12 Months ]
    Steady-state cysteamine-trough WBC cystine levels 30 minutes post RP103 dose at each study visit.
  • Long-Term Safety and Tolerability [ Time Frame: 12 months minimum ]
    The safety profile of RP103 will be investigated with the following assessments: physical exam, vital signs, ECG, clinical laboratory testing and adverse events.


Current Secondary Outcome:

  • Long-Term Safety and Tolerability [ Time Frame: 12 months minimum ]
    The safety profile of RP103 will be investigated by changes from the last study visit as noted in the following parameters: physical examination, vital signs, ECG and clinical laboratory testing. Adverse events (including attribution of treatment-emergent non-serious and serious adverse events) will be recorded.
  • Pharmacokinetic Assessment (Cmax, maximum concentration) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of Cmax.
  • Pharmacokinetic Assessment (Tmax, time to maximum concentration) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of Tmax.
  • Pharmacokinetic Assessment (AUC, area under the curve) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of AUC.


Original Secondary Outcome:

Information By: Horizon Pharma USA, Inc.

Dates:
Date Received: December 5, 2012
Date Started: December 19, 2012
Date Completion:
Last Updated: April 11, 2017
Last Verified: April 2017