NCT05598333进行中（未招募）2 期
A Phase 2, Adaptive, Double-blinded, Placebo Controlled, Randomized, Multi-center Trial to Evaluate the Efficacy, Safety and Tolerability of Intracoronary Infusion of AB-1002 in Adult Subjects With New York Heart Association (NYHA) Class III Heart Failure and Non-ischemic Cardiomyopathy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- AskBio Inc
- 入组人数
- 150
- 试验地点
- 96
- 主要终点
- Cardiovascular Related Death
研究概览
简要总结
This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF.
Subjects will be randomized into one of three treatment groups in a 1:1:1
详细描述
Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 7.15E13vg (n=30-50), 1.43E14 (n=30-50) or placebo group (n=30-50).
Approximately 90 to 150 subjects will be randomly assigned to study intervention Placebo
Study duration until the primary analysis at 52 week will be approximately 37 months including 25 months of recruitment and 52-week Observation Period after dosing. Once all the subjects complete the 52 weeks Observation Period, the treatment groups will be unblinded and primary analysis performed. Study participation duration: The study will last 52 weeks from dosing, with another 4 years of long-term follow-up for a total of 5 years. During the 4 year long-term follow up sites will contact subjects twice a year for two years, then once a year for the remaining two years for safety, efficacy assessments, and survival
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
The IWRS will be programmed with blind-breaking instructions. In case of an emergency, the investigator has the responsibility for determining if unblinding of a subject's intervention assignment is warranted. If the investigator is unavailable, and a treating physician not associated with the study requests emergency unblinding, the emergency unblinding requests are forwarded to the emergency medical advice 24 hours/7 day service.
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
- •Subject must be age ≥18 years of age, at the time of signing the informed consent.
- •Chronic non-ischemic cardiomyopathy
- •15% ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening
- •6MWT >50 meters
- •Medically stable, NYHA Class III HF for a minimum of 4 weeks while on appropriate medical therapy (defined below) including, but not limited to:
- •Beta blocker therapy and 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 (Zareba et al 2011), 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.
- •Women 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 and should also be advised of the benefit for a female partner to use a highly effective method of contraception as a condom may break or leak when having sexual intercourse with a woman of childbearing potential who is not currently pregnant 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
- •Appropriate candidate for protocol-specified intracoronary infusion in the judgment of the infusing interventional cardiologist
排除标准
- •Subjects are excluded from the study if any of the following criteria apply:
- •Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
- •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 screening
- •Uncorrected 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 in judgement of investigator.
- •Known hypersensitivity to contrast dyes (not easily controlled by antihistamines) 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 intra-coronary infusion 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 known to be 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.
- •Chronic Kidney Disease Stage 5, dialysis dependent or eGFR<15 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 of another investigational drug administration prior to administration of AB-1002 that may impact the therapeutic potential of AB-
- •Pregnancy or breastfeeding or plans to become pregnant within the next 12 months at the time of screening
- •Subjects with any other condition which in the opinion of the investigator would preclude participation in the study (including risk for non-compliance and any intercurrent conditions that pose an undue medical hazard, or which could interfere with the interpretation of the study results)
- •Malignant neoplasm within 5 years of dosing, with the exception of those with negligible risk of metastasis or death (such as adequately treated carcinoma in situs of the cervix, basal or squamous cell skin cancer, localized prostate cancer or ductal carcinoma in situ)
- •Any documented history of non-compliance with medications, illicit drug use or laboratory evidence of illicit drug use during screen period
研究组 & 干预措施
Treatment Group 1 AB-1002
Experimental
Randomized in 1:1:1 into one of three groups. Group 1: 7.15E13vg/subject (n=30-50)
干预措施: AB-1002 (Drug)
Treatment Group 2 AB-1002
Experimental
Randomized in 1:1:1 into one of three groups.
Group 2: 1.43E14vg/subject (n=30-50)
干预措施: AB-1002 (Drug)
Treatment Group 3
Placebo Comparator
Randomized in 1:1:1 into one of three groups.
Group 3: Placebo (n=30-50)
干预措施: AB-1002 (Drug)
结局指标
主要结局
Cardiovascular Related Death
时间窗: 52 weeks
NYHA Classification
时间窗: 52 weeks
NYHA Classification Change from baseline
Left Ventricular Ejection Fraction
时间窗: 52 weeks
LVEF change from baseline
Six Minute Walk Test
时间窗: 52 weeks
6MWT distance change from baseline
次要结局
- Heart Failure Related Hospitalizations(52 weeks)
研究者
研究点 (96)
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相关资讯
AskBio Presents Baseline Characteristics from Phase 2 GenePHIT Trial of Umiposgene Parvec in Heart Failure- AskBio presented baseline characteristics from the Phase 2 GenePHIT trial of the investigational gene therapy umiposgene parvec (AB-1002) at the European Society of Cardiology Congress in Munich, Germany.
- GenePHIT randomized more than 170 participants across 64 sites in 12 countries, making it one of the largest gene therapy trials conducted in heart failure to date.
- The enrolled population reflects a representative non-ischemic heart failure with reduced ejection fraction (HFrEF) cohort receiving guideline-directed medical therapy.
- Initial efficacy and safety outcomes from GenePHIT are expected in the first half of 2027.16 days agoAskBio Completes Enrollment in Phase 2 Gene Therapy Trial for Heart Failure- AskBio has completed enrollment of 173 participants in its GenePHIT Phase 2 clinical trial evaluating AB-1002 gene therapy for heart failure with reduced ejection fraction.
- The investigational therapy targets protein phosphatase 1 through a single intracoronary infusion and aims to improve symptoms and survival in patients with non-ischemic cardiomyopathy.
- Initial results from the randomized, placebo-controlled trial are expected in the first half of 2027, representing a significant milestone for addressing the 64 million people worldwide living with heart failure.
- Previous Phase 1 data published in Nature Medicine demonstrated preliminary safety and efficacy, supporting advancement to this larger Phase 2 study.5 months ago