NCT03933410终止2 期
A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of Care
Kaleido Biosciences19 个研究点 分布在 7 个国家目标入组 13 人开始时间: 2019年9月17日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 13
- 试验地点
- 19
- 主要终点
- Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment.
研究概览
简要总结
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
详细描述
We expect the trial to enroll approximately 24 Urea Cycle Disorder (UCD) patients on standard of care with elevated ammonia levels. The planned treatment duration is eight weeks, with a primary endpoint of proportion of subjects who achieve a ≥15% reduction from baseline in fasting ammonia at the end of treatment. Patients will also be followed for safety and tolerability. This clinical trial is intended to allow us to evaluate efficacy of KB195 in reducing ammonia in UCD patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent and willing to comply with protocol-specified procedures.
- •Has any confirmed UCD other than N-acetyl glutamatesynthase (NAGS) deficiency.
- •Is male or female, 12 to 70 years of age (inclusive)
- •If ≥ 18 years old, has a BMI ≥20.0 and < 40.0 kg/m
- •If < 18 years old, has a BMI between 5th percentile and 95th percentile and weight greater than 5th percentile according to age, sex and regionally appropriate growth chart
- •Has evidence of poorly controlled disease on the current standard of care (SOC)
- •If NBT is part of SOC, is on a stable dose and regimen for at least 4 weeks before Screening and the dose is expected to remain stable during the study
- •Is willing to maintain a stable diet throughout the course of study and is willing to continue usual exercise routine.
- •If taking probiotics or prebiotics, is on a stable dose regimen for at least 4 weeks before Screening and the dose and regimen are expected to remain stable during the study
- •Has a negative urine screen for drugs of abuse at Screening
- •If male or female of child bearing potential, agree with use effective method of contraception for the duration of the study and 90 days after last dose of study product
排除标准
- •Is at a high risk for metabolic decomposition.
- •Has had a substantive change in diet or any other aspect of UCD management within 4 weeks before the Screening Visit
- •Has used a systemic anti-infective within 4 weeks before the Screening Visit, or use is anticipated during the study
- •Has been diagnosed with Citrullinemia Type II
- •Is receiving any systemically administered immunosuppressant medication on a chronic basis
- •Has changed the use of or dose of any drug or other compound to modulate GI motility within 4 weeks before the Screening Visit, or the use or dose is expected change during the course of the study
- •Has a history of or active GI or liver disease
- •Has a prior solid organ transplantation including liver transplantation, or is anticipated to receive a liver transplant during study participation
- •Has used an investigational drug, product, or device within 30 days before the Screening Visit
- •Has a contraindication, sensitivity, or known allergy to the study drug
- •Is considered, in the opinion of the PI, to likely be a poor attendee or unlikely for any reason to be able to comply with the study drug procedures
研究组 & 干预措施
KB195
Experimental
KB195 is a novel glycan
干预措施: KB195 (Drug)
结局指标
主要结局
Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment.
时间窗: Day -1 to Day 55
次要结局
- Number of subjects experiencing severe adverse events (SAEs)(Day -28 to Day 84)
- Change from baseline to end of treatment in Gastrointestinal Tolerability Questionnaire (GITQ) scores(Day -28 to Day 84)
- Proportion of subjects normalizing their fasting plasma ammonia concentrations from above the upper limit of normal at baseline to below the upper limit of normal at the end of treatment.(Day -1 to Day 55)
- Number of subjects experiencing adverse events (AEs)(Day -28 to Day 84)
- Change from baseline to end of treatment in Bristol Stool Scale (BSS) scoring.(Day -28 to Day 84)
研究者
研究点 (19)
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