AB-1002 in Patients With Class III Heart Failure

Completed · Phase 1

Conditions studied: Congestive Heart Failure, Heart Failure, Heart Disease, Ischemic, Cardiovascular Diseases, Heart Failure, Systolic, Heart Failure，Congestive, Heart Arrhythmia, Heart Failure, Diastolic, Heart; Complications

In brief

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).

Key facts

Study ID

NCT04179643

Run by

AskBio Inc

People needed

11

Starts

2019-11-20

Expected to finish

2026-07-23

Last updated by the study team

2026-08-06

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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-101.
• Pregnancy or breastfeeding at the time of screening

Where it is running

• Minneapolis Heart Foundation Institute — Minneapolis, Minnesota, United States
• The Linder Center for Education and Research at The Christ Hospital — Cincinnati, Ohio, United States
• The Ohio State University — Columbus, Ohio, United States
• University of Wisconsin at Madison — Madison, Wisconsin, United States

Full record on ClinicalTrials.gov

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