Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
|Study Design:||Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
|Official Title:||An Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With Cystinosis|
- 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.
- 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.
|Actual Study Start Date:||December 19, 2012|
|Study Completion Date:||December 13, 2016|
|Primary Completion Date:||December 13, 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)
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01744782
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo|
|Sao Paulo, SP, Brazil|
|Study Director:||Evelyn Olson, BS||Horizon Pharma USA, Inc.|