NCT04179643已完成1 期
Phase 1 Open Label, Dose Escalation Trial of Intracoronary Infusion of AB-1002 in Subjects With NYHA Class III Heart Failure
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- AskBio Inc
- 入组人数
- 11
- 试验地点
- 4
- 主要终点
- Observed and change from baseline in Peak VO2
研究概览
简要总结
This is a Phase 1, prospective, multi-center, open-label, sequential dose escalation study to explore the safety, feasibility, and efficacy of a single intracoronary infusion of AB-1002 in patients with NYHA Class III heart failure. 11 participants with non-ischemic cardiomyopathy were enrolled. All participants were followed until 12 months post treatment intervention, and then underwent long-term follow-up via semi-structured telephone questionnaires every 6 months for an additional 24 months (+/- 30 days).
详细描述
The safety endpoints will be assessed over the 12-month follow-up period
- Adverse Events
- Clinical laboratory tests
- Vital signs
- Electrocardiograms
Efficacy Endpoints
The efficacy endpoints assessed from baseline to 6 and 12 months will include the following:
- Observed and changes from baseline in peak VO2 assessed by cardiopulmonary exercise testing
- Observed and changes from baseline in 6-minute walk test
- Observed and changes from baseline in NYHA Classification
- Total number of days alive out-of-hospital
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- •Age >18 years of age
- •Chronic non-ischemic cardiomyopathy
- •LVEF 15% ≤ 30% by transthoracic echocardiography (TTE) within 6 months prior to enrollment
- •NYHA Class III HF for a minimum of 3 months HF despite appropriate medical therapy (defined below):
- •Treatment with appropriate HF therapy as tolerated, including, but not limited to:
- •Beta blocker therapy and angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) or sacubitril/valsartan combination therapy (Entresto) for ≥ 90 days prior to enrollment. May also receive aldosterone antagonist therapy. Doses of the above medications must be stable for ≥ 30 days prior to enrollment; and
- •Cardiac resynchronization therapy (CRT), if clinically indicated, must have been implanted ≥ 90 days prior to enrollment. Internal cardioverter defibrillator (ICD) must be implanted, if clinically indicated ≥ 30 days prior to enrollment
- •Females of childbearing potential must use at least one of the following acceptable birth control methods throughout the study and for 6 months after IP administration:
- •Surgically sterile (bilateral tubal ligation, hysterectomy, bilateral oophorectomy) 6 months minimum prior to IP administration
- •Intrauterine device in place for at least 90 days prior to receiving IP
- •Barrier methods (diaphragm plus spermicide or condom) starting at least 30 days prior to receiving IP
- •Abstinence (the subject must be willing to remain abstinent from screening to 6 months after receiving IP). Females are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject
- •Surgical sterilization of the partner(s) (vasectomy) for >180 days prior to IP administration
- •Hormonal contraceptives starting > 90 days prior to IP. If hormonal contraceptives are started less than 90 days prior to receiving IP, subjects must agree to use a barrier method (diaphragm plus spermicide or condom) from screening through 90 days after initiation of hormonal contraceptives
- •Males subjects capable of fathering a child:
- •Must agree to use a condom from IP administration through 6 months after the time of IP administration
- •Must agree not to donate sperm for 6 months after time of receiving IP
- •Documented evidence of vasectomy in males for 180 days minimum prior to receiving IP is an acceptable form of contraception
- •Males who claim abstinence as their method of contraception are allowed provided they agree to use barrier methods should they become sexually active from screening through 6 months after receiving IP. Males are allowed to claim abstinence as their method of contraception only when it is the preferred and usual lifestyle of the subject
- •Ability to sign Informed Consent Form (ICF) and Release of Medical Information Form
- •Appropriate candidate for protocol-specified intracoronary infusion in the judgment of the infusing interventional cardiologist
- •Cohort 3: medical history documentation of PLN-R14Del mutation and an ICD in situ (at least 30 days prior to enrollment)
排除标准
- •Chronic ischemic cardiomyopathy
- •Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment
- •Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
- •Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to enrollment
- •Third degree heart block
- •Clinically significant myocardial infarction (MI) in the judgment of the subject's physician (e.g., ST elevation MI [STEMI] or large non-STEMI) within 6 months prior to enrollment
- •Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), surgically implanted LVAD or cardiac shunt
- •Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LV reduction surgery, heart transplant, conventional revascularization procedure, or valvular repair within 3 months of IP dosing
- •Known hypersensitivity to contrast dyes used for angiography; history of, or likely need for, high-dose steroid pretreatment prior to contrast angiography
- •Expected survival < 1 year in the judgment of the investigator
- •Active or suspected infection within 48 hours prior to enrollment as evidenced by fever or positive culture
- •Known intrinsic liver disease (e.g., cirrhosis, hepatitis A, chronic hepatitis B or hepatitis C virus infection). If serology is positive and PCR is negative, subject may be eligible (confirm with medical monitor).
- •Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], alkaline phosphatase) > 2x upper limit of normal (ULN) within 30 days prior to enrollment.
- •Renal Failure, dialysis dependent or serum creatinine > 2.5 mg/dl within 30 days prior to enrollment
- •Bleeding diathesis or thrombocytopenia defined as platelets <50,000 platelets/μL within 30 days prior to enrollment
- •Anemia defined as hemoglobin <10 g/dL or transfusion dependent within 30 days prior to enrollment
- •Neutropenia defined as absolute neutrophils <1500 mm3 within 30 days prior to enrollment
- •Known AIDS or HIV-positive status, or a previous diagnosis of immunodeficiency with an absolute neutrophil count <1000 cells/mm3
- •Previous participation in a study of gene transfer
- •Receiving investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of another investigational drug administration prior to administration of NAN-101 that may impact the therapeutic potential of NAN-
- •Pregnancy or breastfeeding at the time of screening
研究组 & 干预措施
1.08E14vg AB-1002
Experimental
Intracoronary Infusion of 1.08E14vg AB-1002 to 6 subjects
干预措施: 3 x 10e13vg AB-1002 (Biological)
PLN-R14Del patients: 3.25E13vg AB-1002
Experimental
Intracoronary Infusion of AB-1002 at 3.25E13vg up to 6 subjects with PLN-R14Del genetic mutation
干预措施: 3 x 10e13vg AB-1002 (Biological)
3.25E13vg AB-1002
Experimental
Intracoronary Infusion of 3.25E13vg AB-1002 up to 6 subjects
干预措施: 3 x 10e13vg AB-1002 (Biological)
结局指标
主要结局
Observed and change from baseline in Peak VO2
时间窗: Measured at screening, month 6, 9 and month 12
Cardiopulmonary exercise testing using a modified Bruce protocol
Observed and change from baseline in Echocardiographic assessment in Left Ventricular Ejection Fraction
时间窗: Measured at screening, 18-24 hours post intervention, week 4, Month 3, Month 6 and Month 12
Echocardiography LVEF measurement
次要结局
- Observed and change from baseline in 6-minute walk test distance(Measured at screening, Month 3, Month 6 and month 12)
研究者
研究点 (4)
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