NL-OMON43669已完成3 期
A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - RP103-07
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 12 至 99（—）
入选标准
- •1. Male or female with a documented diagnosis of cystinosis.
- •2. On a stable dose of Cystagon® for at least 21 days prior to Screening.
- •3. WBC cystine level > 1 nmol * cystine/mg of protein on average over at least 2 measurements
- •collected during the 2 years prior to Screening.
- •4. 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.
排除标准
- •1. Younger than 12 years of age.
- •2. 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.
- •3. 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.
- •4. Known hypersensitivity to cysteamine and penicillamine.
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