A Study to Test the Safety of the Study Drug Fosmanogepix and How the Body Handles It in Adults With Severe Kidney Failure on Dialysis
A Phase 1, Non-randomized, Open-label, Multi-center Study to Assess the Pharmacokinetics, Safety, and Tolerability of Intravenous Fosmanogepix in Adult Subjects With End-stage Renal Disease Requiring Intermittent Hemodialysis
This study will evaluate how fosmanogepix is processed by the body (pharmacokinetics, PK) in participants with kidney failure (end-stage renal disease (ESRD) requiring intermittent hemodialysis (IHD) compared with participants with normal kidney function (control group). Fosmanogepix is given by intravenous infusion and is converted in the body to manogepix, the active form of the drug. Fosmanogepix is an investigational and has not been approved by the United States Food and Drug Administration (FDA) or any other regulatory authority for use in humans.
The study will also evaluate the effect of hemodialysis on manogepix levels in participants with ESRD.
The safety and tolerability of fosmanogepix will also be assessed in participants with kidney failure and in matched participants with normal kidney function.
Participation in the study will last up to 11 weeks and will include up to two study periods. Participants with ESDR requiring IHD will receive fosmanogepix in two study periods. Healthy participants will receive fosmanogepix in one study period.
The study will be conducted at two study sites in the United States.
研究概览
研究类型
注册 (估计的)
阶段
- 阶段1
扩展访问
联系人和位置
学习联系方式
- 姓名:Thomas Kaindl, MD
- 电话号码:+41615671505
- 邮箱:thomas.kaindl@basilea.com
学习地点
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Florida
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Orlando、Florida、美国、32809
- 招聘中
- Orlando Clinical Research Center
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Tampa、Florida、美国、33603
- 招聘中
- Genesis Clinical Research
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-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- Body mass index of 18 to 42 kg/m2, inclusive, and a total body weight > 50 kg at Screening.
- Subjects with normal renal function only: Estimated glomerular filtration rate (eGFR) ≥ 90 mL/min
- Subjects with ESRD requiring IHD only: Hemodialysis treatment for at least 3 months prior to Screening, with dialysis performed ≥ 3 times per week and adequate dialysis efficiency as established by document Kt/V ≥ 1.2 on current regimen. The clinical status of the underlying ESRD should be stable
Exclusion Criteria:
- Subjects who are pregnant, breastfeeding, or plan to become pregnant during the study.
- Laboratory abnormality or other medical or psychiatric condition, including recent (within the past year) or active suicidal ideation/behavior, that may increase the risk to study participation, or in the Investigator's judgment, make the subject inappropriate for the study.
- History or presence of clinically-significant disorders, which, in the opinion of the Investigator, are capable of significantly altering the distribution, metabolism, or elimination of the study drug.
- Renal or liver transplant recipients
- Hepatic impairment not related to ESRD
- Use of any prohibited medication within five half-lives plus 14 days prior to Day -1
Subjects with ESRD requiring IHD only:
-Not on stable dosing regimen (i.e., < 3 weeks prior to Screening) of prescription/ non prescription drugs (including dietary/herbal supplements) for the treatment of a stable concomitant disease
学习计划
研究是如何设计的?
设计细节
- 主要用途:其他
- 分配:非随机化
- 介入模型:并行分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:Period 1 and Period 2
Participants with end stage renal disease (ESRD) requiring intermittent hemodialysis (IHD) |
Two doses of intravenous fosmanogepix will be administered on Day 1 and Day 2. Two doses of intravenous fosmanogepix will be administered on Day 1 and Day 2 in each period (Period 1 and 2) |
|
实验性的:Period 1
Participants with normal renal function (control group) |
Two doses of intravenous fosmanogepix will be administered on Day 1 and Day 2. Two doses of intravenous fosmanogepix will be administered on Day 1 and Day 2 in each period (Period 1 and 2) |
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Maximum Plasma Concentration (Cmax) of manogepix
大体时间:Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
Cmax of manogepix will be assessed in participants with ESRD in Period 2 and compared with that of healthy participants in Period 1. |
Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
|
Area under the concentration-time curve (AUC) of manogepix
大体时间:Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
AUC of manogepix will be assessed in participants with ESRD in Period 2 and compared with that of healthy participants in Period 1. |
Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
|
Impact of IHD on the AUC of manogepix (total and unbound) in Periods 1 and 2
大体时间:Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
Impact of IHD on the AUC of manogepix (total and unbound) in Periods 1 and 2 will be assessed after fosmanogepix IV administration in subjects with ESRD requiring IHD. |
Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
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Impact of IHD on the Tmax of manogepix (total and unbound) Periods 1 and 2
大体时间:Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
Impact of IHD on the Tmax of manogepix (total and unbound) will be assessed in Periods 1 and 2 after fosmanogepix IV administration in subjects with ESRD requiring IHD. |
Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
|
Impact of IHD on the Cmax of manogepix (total and unbound) Periods 1 and 2
大体时间:Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
Impact of IHD on the Cmax of manogepix (total and unbound) will be assessed in Periods 1 and 2 after fosmanogepix IV administration in participants with ESRD requiring IHD. |
Blood sampling in Period 1: At Day 1, 2, 3, 4, 5 and 6. Blood sampling in Period 2: At Day 1, 2, 3, 4, 5, 6, 8, 11, 13 and 15.
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Number of participants reporting treatment emergent adverse events (TEAEs)
大体时间:From screening to up to 35 days after last study drug administration.
|
To evaluate the safety and tolerability of fosmanogepix in participants with ESRD requiring IHD, and in healthy participants. |
From screening to up to 35 days after last study drug administration.
|
合作者和调查者
调查人员
- 研究主任:Thomas Kaindl, MD、Basilea Pharmaceutica International Ltd, Allschwil
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- FMGX-CP-111
- 75A50124C00033 (其他赠款/资助编号:BARDA)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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