Safety/Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
This trial is active, not recruiting.
|Sponsor||Raptor Pharmaceuticals Inc.|
|Start date||December 2012|
|End date||November 2016|
|Trial size||12 participants|
|Trial identifier||NCT01744782, RP103-08|
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 However, a subject may withdraw consent or be withdrawn from the study even prior to completing 12 months of participation, e.g., if the Sponsor terminates development of RP103 for cystinosis.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.
|United States||No locations recruiting|
|Other Countries||No locations recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
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.
White Blood Cell (WBC) Cystine Levels
time frame: 12 Months
Long-Term Safety and Tolerability
time frame: 12 months minimum
Male or female participants up to 6 years old.
Inclusion Criteria: - Male or female with a documented diagnosis of cystinosis - No clinically significant change in liver function tests, i.e. 1.5 times ULN for ALT and AST, and/or 1.5 times ULN for total bilirubin, within 6 months prior to Screening - No clinically significant change in renal function, i.e. estimated GFR within 6 months prior to Screening - Must have an estimated GFR > 20 mL/minute/1.73m2 (using the equation from Schwartz 2009 J Am Soc Nephrol 20:629-647) - Female subjects who are sexually active and of childbearing potential, i.e. not surgically sterile (tubal ligation, bilateral oophorectomy, or hysterectomy) or at least 2 years naturally postmenopausal must agree to use an acceptable form of contraception from Screening through completion of the study. Acceptable forms of contraception for this study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to Screening, barrier (spermicidal condom or diaphragm with spermicide), IUD, or a partner who has been vasectomized for at least 6 months - Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study - Has not taken any form of cysteamine in the past Exclusion Criteria: - Subjects with current history of the following conditions or any other health issues that make it, in the opinion of the investigator, unsafe for study participation: - Inflammatory bowel disease if currently active, or prior resection of the small intestine - Heart disease (e.g., myocardial infarction, heart failure, unstable arrhythmias, or poorly controlled hypertension) within 90 days prior to Screening - Active bleeding disorder within 90 days prior to Screening - History of malignant disease within 2 years prior to Screening - Hemoglobin level of < 10 g/dL at Screening or, in the opinion of the investigator, a hemoglobin level that would make it unsafe for study participation - Known hypersensitivity to penicillamine - Female subjects who are nursing, planning a pregnancy, or are known or suspected to be pregnant - Subjects who, in the opinion of the investigator, are not able or willing to comply with study requirements - Has received a kidney transplant or is currently on dialysis - Is 6 years of age or older at the time of the Screening visit [NB: There is no lower age limit.]
|Official title||An Open-Label Safety and Effectiveness Study of Cysteamine Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis|
|Principal investigator||Craig B. Langman, MD|
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