An Open Label Pilot Study to Evaluate the Effect of KB195, a Novel Prebiotic Gluco-Oligosaccharide Mixture, in the Diet of Patients With Urea Cycle Disorders on Gut Nitrogen Metabolism
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Change in labelled (15N-urea) and total urea excretion in urine
研究概览
简要总结
This clinical food study aims to explore the effect of KB195, a novel mixture of oligosaccharides, on the metabolism of nitrogen by the microbiome in patients with urea cycle disorders (UCDs). This will be done using a stable isotope to assess nitrogen metabolism in the blood, urine, and stool. The study will also assess the safety and tolerability of KB195 in patients with UCDs.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed UCD patient at any age ≥ 14 years
- •Parental/legal guardian permission or patient's written informed consent or assent, as applicable
- •Be willing and able to comply with study requirements
排除标准
- •Any medical condition unrelated to the sequelae of UCD
- •Patient has N-acetylglutamate synthase (NAGS) deficiency
- •Recent hospitalization or risk for metabolic decompensation
- •Liver transplantation
- •Systemic antibiotics, probiotics or prebiotics that do not align with study guidances
- •Change in dose or frequency of any drug or other compound to modulate GI motility
- •Contraindications or known allergy/sensitivity to the use of the study products
- •Use of an investigational drug, product or device within 30 days prior to Screening Visit, or current enrolment in another investigational drug, product, or device study
- •Considered to be at risk for noncompliance or unlikely for any reason to be able to comply with the study procedures
结局指标
主要结局
Change in labelled (15N-urea) and total urea excretion in urine
时间窗: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-urea) and total urea excretion in urine
Change in labelled (15N-ammonia) and total ammonia excretion in urine
时间窗: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-ammonia) and total ammonia excretion in urine
Change in labelled (15N-nitrogen) and total nitrogen excretion in urine
时间窗: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-nitrogen) and total nitrogen excretion in urine
次要结局
- Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires(Baseline to Day 32)
- Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires(Baseline to Day 32)