A Phase 2A, Investigator & Subject Blinded, Sponsor Unblinded, Placebo-Controlled, Clinical Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics of CRD-4730 in Participants With Catecholaminergic Polymorphic Ventricular Tachycardia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 7
- 试验地点
- 22
- 主要终点
- Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
This is a Phase 2, multicenter, double-blind, sponsor unblinded, placebo-controlled, single-dose clinical study of CRD-4730 to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of CRD-4730 when administered as single oral doses to participants with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The study will have 2 cohorts in which participants with CPVT will participate in a 3-period, randomized 2-sequence study. Each participant will receive 2 different doses of CRD-4730 and 1 dose of matching placebo, with each study drug administered as a single dose.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Investigator and Subject Blinded, Sponsor Unblinded; Placebo-controlled
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or Females ≥18 years of age, at screening.
- •Confirmed CPVT diagnosis, based on genetic screening for a known RyR2 mutation and a clinical phenotype consistent with CPVT at screening.
- •The participant can perform an EST during which frequent premature ventricular contraction (PVCs) (≥10 per minute), ventricular bigeminy, or higher-grade VA (equivalent to a VA score ≥2) are identified by the investigator.
- •Stable doses of any anti-arrhythmic medication, except amiodarone, for 4 weeks prior to screening.
- •Adhere to all contraceptive criteria.
排除标准
- •Clinically significant structural heart disease, diagnosis of heart failure, or clinically significant coronary artery disease.
- •History of a myocardial infarction, cerebrovascular accident, or transient ischemic attack within 3 months of screening.
- •History of malignancy within the past 5 years at screening (except successfully treated basal cell carcinoma or non-metastatic squamous cell carcinoma of the skin or cervical carcinoma in situ).
- •Female participant that is pregnant or lactating/ breastfeeding, or has plans to do so during the study or within 3 months following last dose of study drug.
- •Use of amiodarone with 3 months prior to screening. NOTE: other protocol defined Inclusion/ Exclusion criteria may apply.
研究组 & 干预措施
Dose 2
CRD-4730 Dose 2 capsule
干预措施: CRD-4730 (Drug)
Dose 3
Placebo capsule to match CRD-4730
干预措施: Placebo (Drug)
Dose 1
CRD-4730 Dose 1 capsule
干预措施: CRD-4730 (Drug)
结局指标
主要结局
Treatment Emergent Adverse Events (TEAEs)
时间窗: Baseline to Day 22
The number of participants with TEAEs including drug-related AEs, serious AEs (SAEs), and AEs leading to study drug discontinuation will be assessed.
Changes in Laboratory Assessments
时间窗: Baseline to Day 15
The number of participants who have normal/ abnormal values at Baseline compared to normal/ abnormal values post-Baseline will be assessed for hematology, serum chemistry and urinalysis.
Changes in Vital Signs Measurement: Systolic and Diastolic blood pressure
时间窗: Baseline to Day 15
Percent change from Baseline to post Baseline will be assessed for systolic and diastolic blood pressure
Changes in Electrocardiogram (ECG) Measurements
时间窗: Baseline to Day 22
Number of participants who have normal/abnormal ECG measurements at Baseline will be compared to normal/abnormal ECG measurement post Baseline
Changes in Vital Signs Measurement: Pulse Rate
时间窗: Baseline to Day 15
Percent change from Baseline to post Baseline will be assessed for pulse rate
Changes in Vital Signs Measurement: Respiratory Rate
时间窗: Baseline to Day 15
Percent change from Baseline to post Baseline will be assessed for respiratory rate
Changes in Vital Signs Measurement: Body Temperature
时间窗: Baseline to Day 15
Percent change from Baseline to post Baseline will be assessed for body temperature
Changes in Physical Exam
时间窗: Baseline to Day 22
General physical exams will be carried out to detect any abnormalities in the cardiovascular, respiratory and other body systems
次要结局
- Change in Ventricular Arrhythmia (VA) score during Exercise Stress Test (EST)(Baseline to Day 15)
- Assessment of PK effect(Baseline through Day 15)