Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
Inclusion Criteria:
1. Subject must be age ≥ 18 years of age, at the time of signing the informed consent.
2. Chronic non-ischemic cardiomyopathy
3. 15% ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening
4. 6MWT > 50 meters
5. Medically stable, NYHA Class III HF for a minimum of 4 weeks while on appropriate
medical therapy (defined below) including, but not limited to:
1. 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
2. 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.
6. 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.
7. 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.
8. Appropriate candidate for protocol-specified intracoronary infusion in the judgment of
the infusing interventional cardiologist.
Exclusion Criteria:
Subjects are excluded from the study if any of the following criteria apply:
9. Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease.
10. Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous
cardiac assist device therapy within 30 days prior to enrollment.
11. Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease,
amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic
LV aneurysm.
12. Cardiac surgery or percutaneous coronary intervention (PCI) within 30 days prior to
screening.
13. Uncorrected Third degree heart block.
14. 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.
15. Prior heart transplantation, left ventricular reduction surgery (LVRS),
cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device),
surgically implanted LVAD or cardiac shunt.
16. 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.
17. 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.
18. Expected survival < 1 year in the judgment of the investigator.
19. Active or suspected infection within 48 hours prior to intra-coronary infusion as
evidenced by fever or positive culture.
20. 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).
21. Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase
[AST], alkaline phosphatase) > 2x upper limit of normal (ULN) within 30 days prior to
enrollment.
22. Chronic Kidney Disease Stage 5, dialysis dependent or eGFR < 15 within 30 days prior to
enrollment.
23. Bleeding diathesis or thrombocytopenia defined as platelets < 50,000 platelets/µL
within 30 days prior to enrollment.
24. Anemia defined as hemoglobin < 10 g/dL or transfusion dependent within 30 days prior to
enrollment.
25. Neutropenia defined as absolute neutrophils <1500 mm3 within 30 days prior to
enrollment.
26. Known AIDS or HIV-positive status, or a previous diagnosis of immunodeficiency with an
absolute neutrophil count < 1000 cells/mm^3.
27. Previous participation in a study of gene transfer.
28. 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-1002.
29. Pregnancy or breastfeeding or plans to become pregnant within the next 12 months at
the time of screening.
30. 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).
31. 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).
32. Any documented history of non-compliance with medications, illicit drug use or
laboratory evidence of illicit drug use during screen period.
Note: Other protocol defined Inclusion/Exclusion Criteria may apply.
Eligibility last updated 10/25/23. Questions regarding updates should be directed to the study team contact.