Clinical Trial: Open-Label Safety & Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cystinosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Long-Term, Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis

Brief Summary:

The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis patients are taking called Cystagon®.

In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of 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. To decide if RP103 is better than Cystagon®, 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.

RP103 is 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®.

Some cystinosis patients have bad breath (halitosis) when they take Cystagon®. Study participants who experience bad breath with Cystagon® will be asked if they would like to participate in an optional "halitosis substudy" to investigate this issue by collecting some extra PK blood samples.


Detailed Summary:
Sponsor: Raptor Pharmaceuticals Inc.

Current Primary Outcome:

  • White Blood Cell (WBC) Cystine Levels [ Time Frame: 7 Months ]
    Superior effectiveness of RP103 vs. Cystagon® will be evaluated comparing WBC cystine levels during two 3-month treatment periods (Cystagon® and RP103). An interim analysis will be performed after 20 subjects complete the two treatment periods; A final analysis will be performed after all 60 subjects have completed.
  • Long-Term Safety and Tolerability [ Time Frame: 7 Months minimum; 24 months maximum ]
    The safety profile of RP103 will be investigated with the following assessments: physical examination, vital signs, ECG, clinical laboratory testing and adverse events.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Quality of Life - General [ Time Frame: 7 months minimum; 24 months maximum ]
    Long-term general quality of life will be assessed using instruments appropriate to the subjects' age and region (US or Europe).
  • Quality of Life - Fatigue/Sleep [ Time Frame: 7 months; 24 months maximum ]
    Long-term quality of life, specifically fatigue/sleep, will be assessed using instruments appropriate to the subjects' age and region (US or Europe).


Original Secondary Outcome: Same as current

Information By: Raptor Pharmaceuticals Inc.

Dates:
Date Received: November 16, 2012
Date Started: January 2013
Date Completion: June 2017
Last Updated: October 3, 2016
Last Verified: October 2016