EUCTR2010-018365-34-NL进行中（未招募）1 期
A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04
Horizon Pharma USA, Inc.0 个研究点目标入组 60 人开始时间: 2010年7月6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
- •1. Male and female subjects must have completed the last visit of Study RP103-03 and be willing to continue with RP103 treatment.
- •OR for patients who did not complete the RP103-03 study:
- •2. Male and female subject with a documented diagnosis of cystinosis.
- •3. Subject must be on a stable dose of Cystagon® at least 21 days prior to Screening.
- •4. Within the last 6 months, no clinically significant change from normal in liver function tests [i.e., 1.5 times ULN for ALT and AST, and/or 1.5 times ULN for total bilirubin] and renal function [i.e., estimated GFR (corrected for body surface area)] at Screening as determined by the Investigator.
- •5. Subject must have an estimated GFR (corrected for body surface area) > 30 mL/minute/1.73 m2.
- •6. Sexually active female subjects of childbearing potential (i.e., not surgically sterile [tubal ligation, hysterectomy, or bilateral oophorectomy] or at least 2 years naturally postmenopausal) must agree to utilize the same acceptable form of contraception from Screening through completion of the Study. The 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, diaphragm with spermicide), IUD, or a partner who has been vasectomized for at least 6 months. For pre-pubescent children, a documented attestation of abstinence from their parent or guardian will be acceptable.
- •7. Subject must be willing and able to comply with the Study restrictions and requirements.
- •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:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Patients enrolled in the previous Study RP103-03 who did not complete their last scheduled study visit or who do not wish to continue on treatment with RP103.
- •AND for patients who did not complete the RP103-03 study:
- •2. Subjects less than 1 year old
- •3. Subjects with current history of the following conditions or any other health issues that make it, in the opinion of the Investigator, unsafe for them to participate:
- •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) 90 days prior to Screening;
- •Active bleeding disorder 90 days prior to Screening;
- •History of malignant disease within the last 2 years.
- •4. Subject with a hemoglobin level of < 10 g/dL at Screening or, in the opinion of the Investigator, a hemoglobin level that would make it unsafe for the subject to participate.
- •5. Subjects with known hypersensitivity to cysteamine and penicillamine.
- •6. Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive serum pregnancy screen.
- •7. Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
研究者
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