A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - Superiority, Safety and Efficacy Study of RP103 in Cystinosis

Horizon Pharma USA, Inc.0 个研究点目标入组 60 人开始时间: 2013年2月26日最近更新: 2018年5月28日

适应症

试验速览

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•Male or female, 12 years and older, with a documented diagnosis of cystinosis.
•(Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study)
•On a stable dose of Cystagon® for at least 21 days prior to Screening.
•WBC cystine level > 1 nmol ½ cystine/mg of protein on average over at least 2 measurements collected during the 2 years prior to Screening.
•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/1.73 m2 (using the equation from Schwartz 2009 J Am Soc Nephrol 20:629-637) 1.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 15
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 45
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•Younger than 12 years of age.
•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 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 < 9 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 cysteamine and 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.

发起方

Horizon Pharma USA, Inc.