Cystinosis Clinical Trial
Official title:
A Long-Term, Open-Label, Safety, Tolerability and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With 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.
n/a
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