Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
A Two-Part, Phase 1, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and WISPER Target Engagement of HTX-001 in Adult Healthy Participants (Part 1) and in Adult Participants With Nonobstructive Hypertrophic Cardiomyopathy (Part 2)
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of HTX-001 in human adult participants.
研究概览
详细说明
HTX-001 is an investigational antisense oligonucleotide (ASO) administered subcutaneously. The purpose of this study is to evaluate safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of multiple doses of HTX-001. This will evaluate multiple doses of HTX-001 in participants with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM) who are receiving standard of care therapy.
研究类型
注册 (估计的)
阶段
- 阶段1
联系人和位置
学习地点
-
-
Massachusetts
-
Boston、Massachusetts、美国、01805
- 招聘中
- Lahey Clinic
-
接触：
- Amanda Kirshkaln-Leahy, MS, ACRP-PM
- 电话号码：781-825-8656
- 邮箱：Amanda.Kirshkaln-Leahy@lahey.org
-
首席研究员：
- Martin Maron, MD
-
-
Missouri
-
Kansas City、Missouri、美国、64111
- 招聘中
- Saint Luke's Health System
-
接触：
- Claire Clinical Research Nurse
- 电话号码：816-932-1627
- 邮箱：claire.pederson@bjc.org
-
首席研究员：
- Erika Hutt Centeno, MD
-
-
Ohio
-
Cincinnati、Ohio、美国、45219
- 招聘中
- The Lindner Research Center at The Christ Hospital
-
接触：
- Kasey Wright
- 电话号码：513-585-1777
- 邮箱：kasey.wright@thechristhospital.com
-
首席研究员：
- Ankit Bhatia,, MD
-
-
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:
- Diagnosis of nHCM
- On a stable regimen of guideline-directed medical therapy for nHCM
- NYHA functional class II or III
- NT-proBNP >300 pg/mL
- Body weight of 50 to 100 kg, inclusive, and BMI ≤40 kg/m2, inclusive
- Adequate renal function
- Not pregnant, and willing to use acceptable birth control methods
- Able and willing to comply with study procedures
Exclusion Criteria:
- Any evidence of left ventricular outflow tract obstruction (LVOTO)
- Implantation of an implantable cardioverter-defibrillator (ICD) within 6 months
- History or current evidence of a clinically significant or uncontrolled underlying condition
- Unable to tolerate subcutaneous dosing
- Any reason that in the opinion of the Investigator would lead to the inability of the participant to comply with the protocol
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：非随机化
- 介入模型：顺序分配
- 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：Open-label evaluation of multiple doses of HTX-001 in participants with symptomatic nHCM
Open label evaluation of multiple doses of HTX-001, across 2-3 dose levels, in participants with symptomatic nHCM who are receiving standard of care therapy |
HTX-001 is an investigational antisense oligonucleotide (ASO) |
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 |
|---|---|---|
|
Safety and Tolerability
大体时间：Through Day 43
|
Adverse events (AEs), serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), and AEs of special interest (AESIs) |
Through Day 43
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Determination of pharmacokinetic parameters
大体时间：Through Day 43
|
Maximum (peak) plasma drug concentration (Cmax) |
Through Day 43
|
|
Determination of pharmacokinetic parameters
大体时间：Through Day 43
|
Time to reach maximum (peak) plasma concentration (Tmax) |
Through Day 43
|
|
Determination of pharmacokinetic parameters
大体时间：Through Day 43
|
Area under the plasma concentration time curve (AUC) |
Through Day 43
|
合作者和调查者
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
- HTX-001-A-101
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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