NCT04134299已完成不适用
An Open-Label 12-Week Food Study to Assess the Safety, Tolerability, And Physiological Effects of An Amino Acid Food Product, AXA4010, In Subjects With Sickle Cell Disease.
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 8
- 主要终点
- Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]
研究概览
简要总结
This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing to participate in the study and provide written informed consent
- •Male and female adolescent (ages 12 to 17 inclusive) and adult (≥ 18 years of age) subjects with a prior medically documented diagnosis of sickle cell disease (SS or β0 thalassemia)
- •For Cohort 2 adult subjects only, a Screening cerebral blood flow/ velocity by transcranial doppler (TCD) should be >110 cm/sec
- •Subjects must show active hemolysis at the time of screening based on each of the following parameters:
- •Lactate dehydrogenase (LDH) ≥ 2x above upper limits of normal (ULN) of the age- and gender-appropriate ranges
- •Indirect bilirubin > 2x of the age- and gender-appropriate ULN
- •Absolute reticulocyte counts > 2x of the age- and gender-appropriate ULN
- •Able to ambulate (without assistance or walkers etc.) in a 6- minute walk test
排除标准
- •Ten (10) or more Vascular-Occlusive Crisis (VOC)s that required hospital or emergency room (ER) visits within the past 12 months
- •Any clinically significant changes abnormalities on the screening 12-lead ECG
- •Estimated glomerular filtration rate <60mL/min/1.73m2 appropriately corrected
- •Hemoglobin (Hb) ≤6.0 g/dL at Screening
- •Subjects on chronic transfusion regimens, or who received a transfusion within the last 2 months prior to screening
- •Experienced VOC or any other sickle crises (eg acute chest syndrome, splenic sequestration, dactylitis, stroke) within the last 2 months prior to Screening
- •Current or history of significant alcohol consumption
- •Other poorly controlled medical conditions as judged by the Investigator
- •Known sensitivity and/or history of clinically significant food intolerance/allergies to proteins (including whey, soy, casein, amino acids, etc.)
- •Unable or unwilling to adhere to contraception requirements
- •Any contraindications to an MRI scan for Cohort 2 only
- •Any other condition that, in the opinion of the Investigator, renders the subject at risk for compliance, compromises the well-being of the subject, or hinders study completion
结局指标
主要结局
Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]
时间窗: Baseline to Week 12
Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
次要结局
- Change Absolute Reticulocyte Count(Baseline to Week 12)
- Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)(Baseline to Week 12)
- Change in Lactate Dehydrogenase (LDH)(Baseline to Week 12)
- Change Indirect Bilirubin(Baseline to Week 12)
研究者
研究点 (8)
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