Open-Label Safety & Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cystinosis
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Long-Term, Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis|
- White Blood Cell (WBC) Cystine Levels [ Time Frame: 7 Months ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]The safety profile of RP103 will be investigated with the following assessments: physical examination, vital signs, ECG, clinical laboratory testing and adverse events.
- Quality of Life - General [ Time Frame: 7 months minimum; 24 months maximum ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]Long-term quality of life, specifically fatigue/sleep, will be assessed using instruments appropriate to the subjects' age and region (US or Europe).
|Study Start Date:||January 2013|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||January 2015 (Final data collection date for primary outcome measure)|
Active Comparator: Cystagon Q6H
From Screening and during Months 1, 2, 3: all subjects will take Cystagon (cysteamine bitartrate) every 6 hours, supplied in 150 and 50mg capsules.
Drug: Cystagon Q6H
Other Name: Cystagon (cysteamine bitartrate)
Experimental: RP103 Q12H
From Months 3.5, 4, 5, 6, 7 and the remainder of participation: all subjects will take RP103 (cysteamine bitartrate delayed-release capsules) every 12 hours, supplied in 75 and 25mg capsules.
Drug: RP103 Q12H
Other Name: RP103 (cysteamine bitartrate delayed-release capsules)
Please refer to this study by its ClinicalTrials.gov identifier: NCT01733316
|United States, California|
|California Pacific Medical Center (CPMC) Research Institute|
|San Francisco, California, United States, 94115|
|Stanford University Medical School|
|Stanford, California, United States, 94305|
|United States, Georgia|
|Emory Children's Center|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60614|
|United States, Texas|
|Baylor College of Medicine / Texas Childrens Hospital|
|Houston, Texas, United States, 77030|
|University Hospital of Leuven|
|Hospices Civils de Lyon|
|Hôpital Robert Debré|
|Ospedale Pediatrico Bambino Gesù|
|Radboud University Nijmegen Medical Center|
|Queen Elizabeth Hospital Birmingham|
|Birmingham, United Kingdom|
|London, United Kingdom|
|Great Ormond Street|
|London, United Kingdom|
|Principal Investigator:||Laurence A Greenbaum, MD, PhD (US)||Emory University|
|Principal Investigator:||Georges Deschênes, MD, PhD (EU)||Hôpital Robert Debré|