EUCTR2017-003851-45-PT进行中（未招募）1 期
A Phase 1/2 Open-label Study in Patients with Arginase 1 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 1 deficiency, with:
- •Hyperargininemia: plasma arginine levels from one or more of 3 baseline samples > 250 µM or 2 of 3 baseline samples > 200 µM (note: 2 of the 3 samples can be collected by home health care personnel)
- •Mutation in the arginase I gene
- •Deficiency in red blood cell (RBC) arginase activity
- •3. Has adequate organ function defined as follows:
- •Bone marrow: Hemoglobin =10 g/dL; WBC >3.0 × 10^9/L; platelet count = 100,000/µL
- •Hepatic (bilirubin): Total bilirubin = 2.0 × upper limit of normal (ULN)
- •Hepatic (transaminases): EITHER
- •Aspartate aminotransferase (AST) and alanine aminotransferase
- •(ALT) = 3.0 × ULN
- •AST and/or ALT > 3.0 × ULN but both = 5.0 × ULN, and in the opinion of the Investigator, related to Arginase 1 deficiency
- •Renal: serum creatinine < 1.5 × ULN
- •4. If female and of child-bearing potential, has a negative serum pregnancy test within 7 days before enrollment
- •5. 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).
- •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 modification) prior to any screening procedures.
- •PART 2 INCLUSION CRITERION:
- •1. Did not experience any adverse event in Part 1 that, in the opinion of the Investigator in consultation with the Sponsor, would preclude continued study 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 RBCs within the 60 days before enrollment
- •2. Currently has an active infection requiring systemic treatment
- •3. Known infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C
- •4. Had unstable hyperammonemia requiring hospitalization within the 14 days before enrollment
- •5. Currently has any comorbid condition or laboratory abnormality 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
- •6. Is currently participating in another therapeutic clinical trial
- •7. Has received any investigational agent within 30 days of enrollment
- •8. Has a history of hypersensitivity to PEG or any other component of the AEB1102 (Co-ArgI-PEG) formulation
- •9. If female, is lactating or breastfeeding
研究者
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