NCT02057341已完成2 期
A Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 12
- 试验地点
- 6
- 主要终点
- Assess the efficacy of the study drug in terms of change from Baseline in 6-minute walk test.
研究概览
简要总结
This is a Phase 2 pilot study, involving a 48-week treatment period, designed to test the effectiveness of investigational study drug ARRY-371797 in treating patients with symptomatic genetic dilated cardiomyopathy due to a lamin A/C gene mutation, and to further evaluate the drug's safety. Approximately 12 patients from the US will be enrolled in this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with idiopathic dilated cardiomyopathy and stable New York Heart Association (NYHA) Class II - IIIa congestive heart failure (CHF).
- •Stable, guidelines-based medical and device therapy, without any CHF hospitalizations or change in heart failure drug dose with ≥ 50% reduction in dose or ≥ 100% increase in dose in the past 3 months.
- •Left ventricular (LV) end diastolic diameter by trans-thoracic echocardiography of > 3.3 cm/m2 (for females) or 3.4 cm/m2 (for males) and/or LV ejection fraction ≤ 45%.
- •Gene positive for a pathogenic mutation in the LMNA gene, as determined by a CLIA-certified clinical laboratory (mutations including but not limited to: splice-site, non-sense, deletion mutations, a mis-sense mutation in a highly conserved codon, a mis-sense mutation involving a major charge change, a mis-sense mutation previously associated with genetic dilated cardiomyopathy).
- •Within 3 weeks prior to first dose of study drug, completed distance during six minute walk test of ≥ 100 m and ≤ 350 m AND/OR ≥ 100 m and ≤ 450 m AND ≤ 60% predicted distance AND patient is symptomatic for dilated cardiomyopathy per Investigator judgment.
- •On the day before and day of first dose of study drug, completed distance during six minute walk test of ≥ 100 m and ≤ 400 m (with the greater value within 10% of the lesser value) AND/OR ≥ 100 m and ≤ 475 m (with the greater value within 10% of the lesser value) AND patient is symptomatic for dilated cardiomyopathy per Investigator judgment.
- •Acceptable hematology, hepatic and renal function laboratory values within 3 weeks prior to first dose of study drug.
- •Additional criteria exist.
排除标准
- •Unstable clinical cardiac symptoms requiring unscheduled hospitalization within 60 days prior to study start.
- •Clinically significant coronary artery disease, as per Investigator judgment.
- •Currently receiving continuous intravenous (IV) inotrope infusion, or presence of a ventricular assist device, or history of prior heart transplantation.
- •Any of the following within 60 days prior to study start: Myocardial infarction, cardiac surgical procedures, acute coronary syndrome, hemodynamically destabilizing cardiac arrhythmia, serious systemic infection with evidence of septicemia, any major surgical procedure requiring general anesthesia.
- •Uncorrected, hemodynamically significant primary valvular disease.
- •Initiation of cardiac resynchronization therapy within 180 days prior to study start.
- •Likelihood, in the Investigator's opinion, of undergoing cardiac transplantation, left ventricular assist device or other device implantation, or other cardiac surgery within the next 6 months; or of requiring continuous IV inotropic treatment, or referral for hospice or end-of-life treatment.
- •Active malignancy (except surgically-curative basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma).
- •Receiving chronic immunosuppressant therapy.
- •Known positive serology for the human immunodeficiency virus (HIV), active hepatitis B and/or hepatitis C.
- •Participation in any other investigational study of drugs or devices within 30 days prior to study start.
- •Additional criteria exist.
研究组 & 干预措施
ARRY-371797 (Dose 1)
Experimental
干预措施: ARRY-371797, p38 inhibitor; oral (Drug)
ARRY-371797 (Dose 2)
Experimental
干预措施: ARRY-371797, p38 inhibitor; oral (Drug)
结局指标
主要结局
Assess the efficacy of the study drug in terms of change from Baseline in 6-minute walk test.
时间窗: 12 weeks
次要结局
- Assess the safety of study drug in terms of adverse events, clinical laboratory tests and electrocardiograms.(48 weeks)
- Assess the efficacy of study drug in terms of left ventricular function.(48 weeks)
- Assess the efficacy of study drug in terms of right ventricular function.(48 weeks)
- Characterize the pharmacokinetics (PK) of study drug and metabolites in terms of plasma concentration-time profiles and model-based PK parameters.(48 weeks)
研究者
研究点 (6)
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