EUCTR2018-003163-67-GB进行中（未招募）1 期
An Open-label, Multicenter Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AEB1102 in Patients with Arginase I Deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 14
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
入选标准
- •A patient must meet all of the following criteria to be enrolled in this study:
- •1) Completed participation in Study CAEB1102-101A without experiencing any clinically significant AE or other unmanageable drug toxicity that precludes continued dosing
- •2) After review of the patient’s data from the CAEB1102-101A study, the Investigator and the Sponsor confirm that it is acceptable for the patient to continue dosing with AEB1102 and that the patient continues to meet the following criteria from the 101A study:
- •a) Is male or female: pediatric patients = 2 to < 18 years of age and adult patients = 18 years of age
- •b) Has documented diagnosis of Arginase I deficiency, with:
- •i) Known mutation in the Arginase I gene
- •ii) Known deficiency in red blood cells (RBCs) arginase activity
- •c) Has adequate organ function defined as follows:
- •i) Bone Marrow: Hemoglobin =10 g/dL; absolute neutrophil count = 1.5x10^9/L; platelet count = 100,000/µL
- •ii) Hepatic: transaminase levels (aspartate aminotransferase/alanine aminotransferase = 2.5-times ULN; total bilirubin = 2.0 mg/dL
- •iii) Renal: serum creatinine < 1.5x ULN
- •3) If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
- •4) If the patient (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation; progesterone-only hormonal contraception associated with inhibition of ovulation; intrauterine device (IUD); or intrauterine hormone-releasing system (IUS).
- •5) Patient or legal guardian is able and willing to provide written informed consent and where required assent, and to comply with all requirements of study participation (including all study procedures and continuation of prescribed diet without modification), prior to any screening procedures
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 9
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 5
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •A patient is excluded from this study if he/she has:
- •1) Clinically significant concurrent disease, serious intercurrent illness, or other extenuating circumstances that, in the opinion of the Investigator, would preclude the patient's participation in the study including:
- •a) Had transfusion of = 2 units of RBC within the 60 days before enrollment
- •b) Currently has an active infection requiring systemic treatment
- •c) Known infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- •d) Currently has uncontrolled hyperammonemia (confirmed ammonia = 100 µmol/L)
- •e) Is currently participating in another therapeutic clinical trial
- •f) Has received any investigational agent (other than pegzilarginase) within 30 days of enrollment
- •g) If female, is lactating or breast feeding
研究者
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