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GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure
EU CTIS ID: 2024-510581-17-00
What this study is testing
evaluate the efficacy and safety of a single antegrade intracoronary artery infusion of AB-1002 compared to placebo infusion in subjects with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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: a. Angiotensin-converting enzyme inhibitor (ACE-I), angiotensin receptor-neprilysin inhibitor (ARNI), angiotensin receptor blocker (ARB), sacubitril/valsartan combination therapy (Entresto), beta blocker therapy, mineralocorticoid receptor antagonist (MRA), sodium-glucose co-transporter 2 inhibitor (SGLT2i) for ≥ 90 days prior to enrollment. Doses of the above medications must be stable for ≥ 30 days prior to enrollment. b. Cardiac resynchronization therapy (Zareba et al., 2011), if clinically indicated, must have been implanted ≥ 90 days prior to enrollment. ICD must be implanted, if clinically indicated ≥ 30 days prior to enrollment.
- Women of childbearing potentia (for definition see also Section 9.4.1)l must use at least one of the following highly effective birth control methods throughout the study: - Intrauterine device in place at least 90 days prior receiving SI - Abstinence (the subject must be willing to remain abstinent from screening to 6 months after receiving SI). 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 SI administration - Hormonal contraceptives associated with the inhibition of ovulation starting > 90 days prior to SI. If hormonal contraceptives are started less than 90 days prior to receiving SI, subjects must agree to use a barrier method (diaphragm plus spermicide or condom) from screening through 90 days after initiation of hormonal contraceptives
You likely can't join if
- Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
- known hypersensivity to SI or to any of the exipients or to the placebo
- Expected survival < 1 year in the judgment of the investigator
- Clinicially relevant infection 48 hours prior to intracoronary infusion
- Known intrinsic liver disease (e.g., cirrhosis, hepatitis A, chronic hepatitis B or hepatitis C virus infection). If serology is positive and polymerase chain reaction (PCR) is known to be negative, the subject may be eligible (confirm with medical monitor)
- Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], alkaline phosphatase or total bilirubin) > 2x upper limit of normal (ULN) within 30 days prior to enrollment. In the case that patients have a confirmed diagnosis of benign liver dysfunction (e.g. Gilbert’s syndrome with unconjugated bilirubin not exceeding 4 mg/dL), patients may be eligible for inclusion into the study).
See the full eligibility criteria
Who can join
- 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: a. Angiotensin-converting enzyme inhibitor (ACE-I), angiotensin receptor-neprilysin inhibitor (ARNI), angiotensin receptor blocker (ARB), sacubitril/valsartan combination therapy (Entresto), beta blocker therapy, mineralocorticoid receptor antagonist (MRA), sodium-glucose co-transporter 2 inhibitor (SGLT2i) for ≥ 90 days prior to enrollment. Doses of the above medications must be stable for ≥ 30 days prior to enrollment. b. Cardiac resynchronization therapy (Zareba et al., 2011), if clinically indicated, must have been implanted ≥ 90 days prior to enrollment. ICD must be implanted, if clinically indicated ≥ 30 days prior to enrollment.
- Women of childbearing potentia (for definition see also Section 9.4.1)l must use at least one of the following highly effective birth control methods throughout the study: - Intrauterine device in place at least 90 days prior receiving SI - Abstinence (the subject must be willing to remain abstinent from screening to 6 months after receiving SI). 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 SI administration - Hormonal contraceptives associated with the inhibition of ovulation starting > 90 days prior to SI. If hormonal contraceptives are started less than 90 days prior to receiving SI, 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 not to donate sperm for 6 months (in Belgium: 12 months) after time of receiving SI - Documented evidence of vasectomy in males for 180 days minimum prior to receiving SI 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 (in Belgium: 12 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
- To protect partners of study subjects from potential viral shedding, sexually active subjects and their partners must agree to use barrier methods of contraception (condoms, female condoms, and dams) for a minimum of 6 months (in Belgium: 12 months) after SI administration; barrier methods should be used regardless of any other applied birth control methods.
What rules you out
- Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
- known hypersensivity to SI or to any of the exipients or to the placebo
- Expected survival < 1 year in the judgment of the investigator
- Clinicially relevant infection 48 hours prior to intracoronary infusion
- Known intrinsic liver disease (e.g., cirrhosis, hepatitis A, chronic hepatitis B or hepatitis C virus infection). If serology is positive and polymerase chain reaction (PCR) is known to be negative, the subject may be eligible (confirm with medical monitor)
- Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], alkaline phosphatase or total bilirubin) > 2x upper limit of normal (ULN) within 30 days prior to enrollment. In the case that patients have a confirmed diagnosis of benign liver dysfunction (e.g. Gilbert’s syndrome with unconjugated bilirubin not exceeding 4 mg/dL), patients may be eligible for inclusion into the study).
- 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 is defined as hemoglobin <10 g/dL or transfusion dependent within 30 days prior to enrollment
- Neutropenia is 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
- Intravenous (IV) inotropic therapy, intra-aortic balloon pump (IABP) or percutaneous cardiac assist device therapy within 30 days prior to enrollment
- 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-1002
- 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 noncompliance 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 noncompliance with medications; illicit drug use or laboratory evidence of illicit drug use during screen period
- Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
- Cardiac surgery or PCI within 30 days prior to enrollment
- Uncorrected third degree heart block
- Clinically significant 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 SI dosing in the judgment of the 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
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.