EUCTR2017-003851-45-GB进行中（未招募）1 期
A Phase 1/2 Open-label Study in Patients with Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
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. Is male or female: Pediatric patients = 2 to < 18 years of age; and adult patients = 18 years of age.
- •2. Has documented diagnosis of Arginase I deficiency, with:
- •Hyperargininemia: plasma arginine levels consistently = 2-times the upper limit of normal (ULN) (> 250 µM)
- •Known mutation in the Arginase I gene
- •Known decrease in arginase activity (< 5%) in red blood cells (RBCs)
- •3. Has adequate organ function defined as follows:
- •a. Bone Marrow: Hemoglobin =10 g/dL; absolute neutrophil count = 1.5x10^9/L; platelet count = 100,000/µL
- •b. Hepatic: transaminase levels (aspartate aminotransferase/alanine
- •aminotransferase = 2.5-times ULN; total bilirubin = 2.0 mg/dL
- •c. Renal: serum creatinine < 1.5x ULN
- •4. If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment.
- •5. If sexually active (male or female), must be surgically sterile, postmenopausal (female), or must agree to use a physician-approved method of birth control during the study and for a minimum of 30 days after the last study drug administration.
- •6. 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.
- •PART 2 INCLUSION CRITERION:
- •1. Did not experience any safety or tolerability event in Part 1 which would preclude continued participation and dosing of AEB1102
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 7
- •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 meets any of the following criteria
- •1. Had transfusion of = 2 units of RBC within the 60 days before enrollment
- •2. Currently has an active infection of any kind requiring systemic treatment
- •3. Known infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- •4. Had severe hyperammonemia requiring hospitalization within the 14 days before enrollment. Had more than 1 episode of hyperammonemia requiring hospitalization within the 30 days prior to enrollment
- •5. Currently has uncontrolled hyperammonemia (ammonia = 100 µmol/L)
- •6. Currently has any co-morbid condition that in the opinion of the investigator might compromise the patient's safety, might interfere with participation in the trial, or might interfere with the interpretation of trial results
- •7. Is currently participating in another therapeutic clinical trial
- •8. Has received any investigational agent within 30 days of enrollment
- •9. Has a history of hypersensitivity to polyethylene glycol (PEG) or any other component of the AEB1102 (Co-ArgI-PEG) formulation
- •10. If female, is lactating or breast feeding
研究者
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