What is the mechanism of action of this treatment?

Common treatments for heart failure include medications like ACE inhibitors, beta-blockers, and diuretics, which work by reducing blood pressure, heart rate, and fluid overload, respectively. Gene therapy and biologic agents, such as the investigational product BNP116.sc-CMV.I1c, aim to improve heart function at a molecular level. These therapies can involve the delivery of genes or proteins that enhance cardiac muscle contractility, reduce fibrosis, or improve cellular energy metabolism. For heart failure patients, these advanced treatments offer the potential for more targeted and effective management of the disease, potentially leading to improved heart function and quality of life.

What side effects are associated with this type of intervention?

Gene therapies and biologic agents for heart failure, such as the investigational BNP116.sc-CMV.I1c, are designed to improve heart function by targeting specific genetic or molecular pathways. Common side effects of these treatments can include immune reactions, such as hypersensitivity or mild lymphadenopathy, as well as potential off-target effects that may impact other organs. Traditional treatments for heart failure, including ACE inhibitors, beta-blockers, and diuretics, often have side effects like hypotension, bradycardia, electrolyte imbalances, and renal dysfunction. It is crucial to monitor patients closely for adverse reactions and adjust treatment as necessary.

What prior FDA approvals exist?

The FDA has approved several treatments for heart failure, including traditional medications like ACE inhibitors, beta-blockers, and diuretics. In recent years, there has been progress in biologic agents and gene therapies aimed at improving heart function. For instance, sacubitril/valsartan (Entresto) is a combination drug that enhances heart function by inhibiting neprilysin and blocking angiotensin II receptors. Additionally, the FDA has approved the use of certain biologic agents like stem cell therapies, which are still largely experimental but show promise in regenerating damaged heart tissue. While specific gene therapies for heart failure are still under investigation, the approval of related biologic treatments indicates a growing interest and potential in this area.

What other types of research exist?

Promising novel alternatives for heart failure treatment in 2024 include: 1) SGLT2 inhibitors (e.g., dapagliflozin, empagliflozin) which have shown significant benefits in heart failure patients with reduced ejection fraction. 2) Vericiguat, a soluble guanylate cyclase stimulator, which has been approved for reducing the risk of cardiovascular death and heart failure hospitalization. 3) Omecamtiv mecarbil, a cardiac myosin activator, which improves cardiac contractility and has shown positive results in clinical trials. 4) Gene therapies targeting specific genetic mutations associated with heart failure, though still in experimental stages, are also a promising area of research.

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Virus Therapy

NAN-101 for Heart Failure (NAN-CS101 Trial)

Phase 1

Recruiting

Research Sponsored by Asklepios Biopharmaceutical, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age >18 years of age

Chronic non-ischemic cardiomyopathy

Must not have

Anemia defined as hemoglobin <10 g/dL or transfusion dependent within 30 days prior to enrollment

Known hypersensitivity to contrast dyes used for angiography; history of, or likely need for, high-dose steroid pretreatment prior to contrast angiography

Timeline

Screening 3 weeks

Treatment Varies

Follow Up measured at screening, 18-24 hours post intervention, week 4, month 6 and month 12

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new heart treatment given directly into the heart's blood vessels. It aims to help patients with severe heart failure who have significant symptoms and limited physical activity. The treatment is designed to improve heart function and quality of life.

Who is the study for?

This trial is for adults over 18 with Class III heart failure who've had symptoms despite treatment for at least 6 months. They must have a left ventricular ejection fraction (LVEF) ≤30% and be able to consent. Women of childbearing age and men must agree to use contraception during the study and for six months after.

What is being tested?

The trial tests a single intracoronary infusion of NAN-101 in patients with symptomatic congestive heart failure, monitoring them for safety, feasibility, and efficacy over a period of up to three years including follow-ups via phone every six months after the first year.

What are the potential side effects?

As this is an early-phase trial primarily assessing safety, specific side effects are not listed but may include typical risks associated with intracoronary procedures such as bleeding or infection, as well as any potential gene therapy-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am older than 18 years.

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I have a long-term heart condition that is not caused by blocked arteries.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have anemia or needed a blood transfusion in the last 30 days.

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I am allergic to dyes used in certain heart and blood vessel imaging tests.

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I am on dialysis or my kidney function test shows creatinine over 2.5 mg/dl.

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I have long-term heart muscle damage from reduced blood flow.

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I have AIDS, am HIV-positive, or have a low neutrophil count.

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I have had heart surgery or a device implanted for heart support.

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I do not have specific heart or thyroid conditions.

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I have not had heart surgery or a heart procedure in the last 30 days.

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I have a diagnosed liver condition.

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I have not had a fever or positive infection test in the last 48 hours.

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My platelet count is above 50,000.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ measured at screening, 18-24 hours post intervention, week 4, month 6 and month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~measured at screening, 18-24 hours post intervention, week 4, month 6 and month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and measured at screening, 18-24 hours post intervention, week 4, month 6 and month 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Observed and change from baseline in Echocardiographic assessment in Left Ventricular Ejection Fraction

Observed and change from baseline in Peak VO2

Secondary study objectives

Observed and change from baseline in 6-minute walk test distance

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: PLN-R14Del patients: 3.25E13vg AB-1002Experimental Treatment1 Intervention

Intracoronary Infusion of AB-1002 at 3.25E13vg up to 6 subjects with PLN-R14Del genetic mutation

Group II: 3.25E13vg AB-1002Experimental Treatment1 Intervention

Intracoronary Infusion of 3.25E13vg AB-1002 up to 6 subjects

Group III: 1.08E14vg AB-1002Experimental Treatment1 Intervention

Intracoronary Infusion of 1.08E14vg AB-1002 to 6 subjects

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for heart failure include medications like ACE inhibitors, beta-blockers, and diuretics, which work by reducing blood pressure, heart rate, and fluid overload, respectively. Gene therapy and biologic agents, such as the investigational product BNP116.sc-CMV.I1c, aim to improve heart function at a molecular level. These therapies can involve the delivery of genes or proteins that enhance cardiac muscle contractility, reduce fibrosis, or improve cellular energy metabolism. For heart failure patients, these advanced treatments offer the potential for more targeted and effective management of the disease, potentially leading to improved heart function and quality of life.

Targeted Genetic Reduction of Mutant Huntingtin Lessens Cardiac Pathology in the BACHD Mouse Model of Huntington's Disease.[Long-term effects of TCV116 on cardiac function changes after myocardial infarction].Cardiac contractility modulation electrical signals improve myocardial gene expression in patients with heart failure.