此页面是自动翻译的，不保证翻译的准确性。请参阅 英文版 对于源文本。

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.

2026年6月10日 更新者：Cajal Therapeutics Inc.

A Phase 1, 3-Part, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Oral Doses of CTX001 in Healthy Adult Participants.

This study is testing CTX001 for certain conditions where the body does not have enough available iron or has difficulty storing or moving iron properly. The purpose of this study is to investigate any side effects that may happen with CTX001, how CTX001 is absorbed by and processed in the body, and how CTX001 affects iron levels in the blood when administered with or without iron and/or food.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

The study drug CTX001 is an investigational treatment for certain diseases where the body does not have enough iron in the bone marrow (the hollow inner parts of bones). Iron is an important part of red blood cells, which are made in the bone marrow. When there is not enough iron in the bone marrow, it can lead to anaemia. Conditions like chronic kidney disease, bone marrow diseases, intestinal diseases, and bleeding disorders all can cause anaemia due to low iron levels, or iron that gets trapped in other body parts like the spleen and cannot get to the bone marrow. Although anemia can sometimes be treated with iron pills or iron infusions, some patients cannot tolerate these treatments and others do not respond to them because either their intestines don't absorb the iron or because the body has improperly stored the iron where it can't be used to make red blood cells. CTX001 is a pill that is swallowed by mouth. Inside the body, CTX001 is expected to bind to iron, help the iron be absorbed by the intestine and help the iron move out of body parts where it might be stuck. If effective, CTX001 could be used to treat anemia in patients with chronic health conditions.

研究类型

介入性

注册 (估计的)

72

阶段

  • 第一阶段早期

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 澳大利亚
    • Victoria
      • Melbourne、Victoria、澳大利亚、3004
        • 招聘中
        • Nucleus Network

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人

接受健康志愿者

是的

描述

Inclusion Criteria:

  • Capable of giving informed consent
  • Agrees to use effective contraception
  • Body Mass Index (BMI) between 18.0 and 32.0 kg/m2
  • Healthy by medical evaluation and medical history
  • Hematological parameters, serum iron, transferrin and ferritin are within normal range and transferrin saturation is within normal range and greater than or equal to 20%
  • Can swallow tablets and has suitable venous access for blood sampling

Exclusion Criteria:

  • Has dietary requirements that may be difficult to accommodate
  • Is a regular user of cannabis or has a history of illicit drug abuse within 1 year
  • Has a history of alcohol abuse or binge drinking within 6 months
  • Is a regular user of tobacco or nicotine-containing products
  • Unwilling or unable to comply with the lifestyle guidelines described in the protocol
  • Has clinically significant history or evidence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurological, immunological, or psychiatric disorder(s) as determined by the Investigator
  • Has any concurrent disease or condition or physical, psychological, mental, and/or social reason that, in the opinion of the Investigator, would make the participant unsuitable for participation in the clinical study
  • Has received a blood transfusion within 1 year
  • Has donated whole blood within 6 months or plasma within 30 days
  • Requires prescription medication or regular use of non-prescription medication
  • Is an employee of the Sponsor, the CRO, or of any organization or site(s) associated with this study, or any immediate family member who is in a dependent relationship with a study site employee who is involved in the conduct of the study

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：顺序分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：Single Ascending Dose (CTX001)
Up to 5 sequential, single ascending dose cohorts.
药品：CTX001 With and Without Iron
There are two treatment periods. In the first, CTX001 is administered as a single dose (tablet) by itself. In the second, CTX001 is administered as a single dose (tablet) with iron.
安慰剂比较：Single Ascending Dose (Placebo)
Up to 5 sequential, single ascending dose cohorts
药品：Placebo With and Without Iron
There are two treatment periods. In the first, placebo is administered as a single dose (tablet) by itself. In the second, placebo is administered as a single dose (tablet) with iron.
实验性的：Food Effect (CTX001)
Up to 3 sequential, single ascending dose food effect cohorts
药品：CTX001 With Iron
There are two treatment periods. In one, CTX001 is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, CTX001 is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
安慰剂比较：Food Effect (Placebo)
Up to 3 sequential, single ascending dose food effect cohorts
药品：Placebo With Iron
There are two treatment periods. In one, placebo is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, placebo is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
实验性的：Multiple Ascending Dose (CTX001)
Up to 3 multiple ascending dose cohorts.
药品：CTX001 Multiple Dose
CTX001 (tablet) is administered daily for 7 consecutive days.
安慰剂比较：Multiple Ascending Dose (Placebo)
Up to 3 multiple ascending dose cohorts
药品：Placebo Multiple Dose
Placebo (tablet) is administered daily for 7 consecutive days.

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
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
大体时间：From first dose of study drug through the last study visit, approximately 15 days.
An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to the study intervention. An AE was considered a treatment-emergent AE (TEAE) if the AE started after initial study drug administration and before the last study visit.
From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Serious TEAEs
大体时间：From first dose of study drug through the last study visit, approximately 15 days.
A serious AE (SAE) is defined as any AE that, at any dose, meets one or more of the following criteria: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital anomaly or birth defect; or any important medical event that may jeopardize the participant or may require medical intervention to prevent one of the outcomes listed above.
From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Physical Examination
大体时间：From the first dose of study drug through the last study visit; approximately 15 days.
Abnormalities in physical examinations were based on investigator's discretion.
From the first dose of study drug through the last study visit; approximately 15 days.
Number of Participants with Clinically Significant Changes in Safety Laboratory Values
大体时间：From first dose of study drug through last study visit, approximately 15 days.
Safety laboratory evaluations included blood counts, serum chemistries, coagulation studies, and urinalyses. Determination of clinical significance was based on investigator discretion.
From first dose of study drug through last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Vital Signs
大体时间：From the first dose of study drug through the last study visit, approximately 15 days.
Vital signs included heart rate, respiratory rate, blood pressure, and temperature. Determination of clinical significance was based on investigator discretion.
From the first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Electrocardiograms
大体时间：From the first dose of study drug through the last study visit, approximately 15 days.
Electrocardiograms are tests that measure the electrical activity of the heart. Determination of clinical significance was based on investigator discretion.
From the first dose of study drug through the last study visit, approximately 15 days.

次要结果测量

结果测量
大体时间
Maximum Observed Plasma Concentration (Cmax) of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Time to Maximum Observed Plasma Concentration (Tmax) of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Area Under the Curve from Time Zero Extrapolated to Infinity (AUC0-inf) of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Area Under the Curve From Time Zero to Time of Last Quantifiable Concentration (AUC0-t) of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Apparent Clearance (CL/F) of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Plasma Half-Life of CTX001
大体时间：Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Iron From Baseline
大体时间：Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Transferrin From Baseline
大体时间：Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Transferrin Saturation From Baseline
大体时间：Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Ferritin From Baseline
大体时间：Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Cajal Therapeutics Inc.

调查人员

  • 首席研究员：Aarthy Joseph, Dr、Nucleus Network

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年6月1日

初级完成 (估计的)

2026年10月1日

研究完成 (估计的)

2026年12月30日

研究注册日期

首次提交

2026年5月5日

首先提交符合 QC 标准的

2026年5月5日

首次发布 (实际的)

2026年5月11日

研究记录更新

最后更新发布 (实际的)

2026年6月12日

上次提交的符合 QC 标准的更新

2026年6月10日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • CTX001-101

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

订阅