Ask-CHF
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StatusAccepting Candidates
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Age18 Years - N/A
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SexesAll
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Healthy VolunteersNo
Objective
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
Description
Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 3.25E13vg (n=30-50), 6.5E13 (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
Details
Full study title | 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 |
Protocol number | OCR45540 |
ClinicalTrials.gov ID | NCT05598333 |
Phase | Phase 2 |
Eligibility
Inclusion Criteria:
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
Exclusion Criteria:
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
Lead researcher
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Mustafa M Ahmed, MDCardiologist (Heart Specialist), Advanced Heart Failure and Transplant Specialist
Participate in a study
Here are some general steps to consider when participating in a research study:
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Step1
Contact the research team
Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.
Primary contact
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Step2
Get screened to confirm eligibility
You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.
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Step3
Provide your consent to participate
If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.
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Step4
Participate
If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.