Safety/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. Thereafter, US subjects will exit the study and transition to the commercially approved drug PROCYSB® just as soon as their insurance/payment details have been arranged. In Brazil, after the minimum 12 months of participation, subjects will transition to an expanded access program and continue to receive the drug at no personal cost. However, a subject may withdraw consent or be withdrawn from the study even prior to completing 12 months of participation, e.g., if the Sponsor terminates development of RP103 for cystinosis.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.
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open-Label Safety and Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis|
- White Blood Cell (WBC) Cystine Levels [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]The safety profile of RP103 will be investigated with the following assessments: physical exam, vital signs, ECG, clinical laboratory testing and adverse events.
|Study Start Date:||December 2012|
|Estimated Study Completion Date:||March 2016|
|Estimated Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Experimental: RP103 Q12H
From Day 1 and throughout the duration of participation, subjects will take RP103 (Cysteamine Bitartrate Delayed-release Capsules) every 12 hours, supplied in 75mg and 25mg capsules.
Drug: RP103 Q12H
Other Name: (Cysteamine Delayed-release Capsules)
Please refer to this study by its ClinicalTrials.gov identifier: NCT01744782
|Contact: Krishna R Polu, MD||1-415-408-6200 ext email@example.com|
|Contact: Daniel Miconi||1-415-408-6200 ext firstname.lastname@example.org|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago||Recruiting|
|Chicago, Illinois, United States, 60614|
|Contact: Heather Price, MS 773-755-6368 email@example.com|
|Principal Investigator: Craig B Langman, MD|
|Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo||Recruiting|
|Sao Paulo, SP, Brazil|
|Contact: Maria Helena Vaisbich, MD 55-11-2661-8833 firstname.lastname@example.org|
|Contact: Juliiana de Oliveria Achili 55-11-2661-8833 email@example.com|
|Principal Investigator:||Craig B. Langman, MD||Ann & Robert H Lurie Children's Hospital of Chicago|