EUCTR2012-002773-64-BE进行中（未招募）1 期
A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis - RP103-07
Raptor Therapeutics Inc.0 个研究点目标入组 60 人开始时间: 2013年4月19日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 60
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Male or female with a documented diagnosis of cystinosis.
- •2. On a stable dose of Cystagon® for at least 21 days prior to
- •3. 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.
- •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.
- •5. No clinically significant change in renal function, i.e.
- •estimated GFR within 6 months prior to Screening.
- •6. Must have an estimated GFR > 20 mL/minute/1.73 m2 (using
- •the equation from Schwartz 2009 J Am Soc Nephrol 20:629-
- •7. Female subjects who are sexually active and of childbearing
- •potential, i.e. not surgically sterile (tubal ligation, bilateral
- •oopherectomy, 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.
- •8. Subject or their parent or guardian must provide written
- •informed consent and assent (where applicable) prior to
- •participation in the study.
- •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
排除标准
- •Exclusion Criteria:
- •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
- •- History of malignant disease within 2 years prior to
- •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.
- •5. Female subjects who are nursing, planning a pregnancy, or
- •are known or suspected to be pregnant.
- •6. Subjects who, in the opinion of the Investigator, are not able
- •or willing to comply with study requirements.
研究者
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