Heart Failure Clinical Trial

NAN-101 in Patients With Class III Heart Failure

Summary

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 BNP116.sc-CMV.I1c in patients with NYHA Class III heart failure. Patients with symptomatic congestive heart failure will be enrolled until up to 12 subjects have received infusions of investigational product. All patients will be followed until 12 months post treatment intervention, and then undergo long-term follow-up via semi-structured telephone questionnaires every 6 months for an additional 24 months (+/- 30 days).

View Full Description

Full Description

The primary endpoint for this study is safety as measured by the following which will be assessed over the 12 month follow-up period as indicated in the Data Collection Table:

Adverse Events
All-cause Mortality
Heart failure (HF) Hospitalization

Secondary Endpoints

The secondary safety endpoints assessed will include the following:

Echocardiographic assessments at 4 weeks +/- 3 days post-administration of BNP116.sc-CMV.I1c including
Echocardiographic assessments of LVEF, LVEVD, LVEDVI, VLESV, LVEVI, SpI and GLSand degree of mitral regurgitation o

The secondary efficacy endpoints will explore efficacy. Functional endpoints will be assessed as changes from baseline to 6 and 12 months following investigational product administration as indicated. These endpoints include:

Functional Status & Hospitalizations

Peak VO2 assessed by cardiopulmonary exercise testing
6-minute walk test
New York Heart Association (NYHA) Classification
Total number of days alive out-of-hospital (as well as total days out-of-hospital as a % of total days alive post study intervention)

Physiologic Assessments at 6 and 12 months compared to baseline

Echocardiographic assessments of LVEF, LVEVD, LVEDVI, VLESV, LVEVI, SpI and GLSand degree of mitral regurgitation
NT-proBNP level

Quality of Life at 6 and 12 months compared to baseline

o Health related quality of life as assessed by Minnesota Living with Heart Failure Questionnaire (MLWHFQ)

The following endpoints will also be measured over the 12 month follow-up period and long-term follow-up period (until month 36 post-intervention):

Survival
Cardiac transplantation
Left ventricular assist device (LVAD) implantation

View Eligibility Criteria

Eligibility Criteria

Inclusion criteria:

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)

Exclusion Criteria:

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

Study is for people with:

Heart Failure

Phase:

Phase 1

Estimated Enrollment:

12

Study ID:

NCT04179643

Recruitment Status:

Recruiting

Sponsor:

Asklepios Biopharmaceutical, Inc.

Check Your Eligibility

Let’s see if you might be eligible for this study.

What is your age and gender ?

Submit

There are 4 Locations for this study

See Locations Near You

Minneapolis Heart Foundation Institute
Minneapolis Minnesota, 55407, United States More Info
Contact
612-863-3818
Jay Traverse, MD
Principal Investigator
The Linder Center for Education and Research at The Christ Hospital
Cincinnati Ohio, 45219, United States More Info
Denise Krabbe, RN
Contact
513-585-1790
[email protected]
Timothy D Henry, MD
Principal Investigator
The Ohio State University
Columbus Ohio, 43210, United States More Info
Contact
614-247-6797
Konstantinos Boudoulas, MD
Principal Investigator
University of Wisconsin at Madison
Madison Wisconsin, 53792, United States More Info
Contact
608-265-0612
David Murray, MD
Principal Investigator

How clear is this clinincal trial information?

Study is for people with:

Heart Failure

Phase:

Phase 1

Estimated Enrollment:

12

Study ID:

NCT04179643

Recruitment Status:

Recruiting

Sponsor:


Asklepios Biopharmaceutical, Inc.

How clear is this clinincal trial information?

×

Introducing, the Journey Bar

Use this bar to access information about the steps in your cancer journey.