NAN-101 for Heart Failure

(NAN-CS101 Trial)

Recruiting at3 trial locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: AskBio Inc

Must be taking: Beta blockers, ACE inhibitors

Disqualifiers: Ischemic cardiomyopathy, Cardiac surgery, others

No Placebo Group

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that your current heart failure medications, like beta blockers and ACE inhibitors, be stable for at least 30 days before joining. It doesn't specify stopping other medications, so you should discuss your specific situation with the trial team.

What evidence supports the effectiveness of the drug NAN-101 for heart failure?

Research shows that B-type natriuretic peptide (BNP), which is similar to nesiritide, is effective in treating heart failure by helping to reduce pressure in the heart and improve symptoms. Nesiritide, a synthetic form of BNP, has been shown to be effective in clinical trials for heart failure, suggesting that similar treatments like NAN-101 may also be beneficial. ^{1 2 3 4 5}

What makes the drug NAN-101 unique for treating heart failure?

NAN-101 is unique because it involves the use of natriuretic peptides, which are hormones that help regulate blood pressure and fluid balance, and have potential therapeutic effects in heart failure by counteracting harmful hormonal systems. This approach is different from traditional treatments that primarily focus on suppressing these systems. ^{1 6 7 8 9}

Research Team

Eligibility Criteria

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.

Inclusion Criteria

I am older than 18 years.

Your heart's pumping ability is very low, as measured by a recent heart ultrasound.

I am a man who can father a child and agree to use a condom for 6 months after treatment.

Exclusion Criteria

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

I haven't taken part in another clinical study or taken any investigational drugs within the last 30 days.

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

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Single intracoronary infusion of AB-1002

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Multiple visits (in-person and virtual)

Long-term follow-up

Participants undergo long-term follow-up via semi-structured telephone questionnaires

24 months

Every 6 months (virtual)

Treatment Details

Interventions

• NAN-101 (Virus Therapy)

Trial Overview 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.

Participant Groups

Group I: PLN-R14Del patients: 3.25E13vg AB-1002 Experimental Treatment 1 Intervention

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

Group II: 3.25E13vg AB-1002 Experimental Treatment 1 Intervention

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

Group III: 1.08E14vg AB-1002 Experimental Treatment 1 Intervention

Intracoronary Infusion of 1.08E14vg AB-1002 to 6 subjects

Who Is Running the Clinical Trial?

AskBio Inc

Lead Sponsor

Trials

8

Recruited

440+

Asklepios Biopharmaceutical, Inc.

Lead Sponsor

Trials

8

Recruited

440+

Findings from Research

B-type natriuretic peptide (BNP) and its precursor nt-proBNP are effective biomarkers for diagnosing heart failure, particularly useful in emergency settings to determine the cause of breathlessness.

Nesiritide, a recombinant form of BNP, is being investigated as a potential treatment for patients with acutely decompensated heart failure, highlighting ongoing research into BNP's therapeutic applications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of b-type natriuretic peptide in heart failure management. Newton, PJ., Betihavas, V., Macdonald, P.[2015]

B-type natriuretic peptide (BNP) shows promise as a diagnostic and therapeutic tool for heart failure, but more research is needed to understand its variability in patients over time.

Nesiritide, a new intravenous treatment for acute heart failure, effectively acts as a vasodilator and may improve long-term outcomes, although its role in chronic therapy for end-stage patients is still uncertain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of B-type natriuretic peptide in heart failure. Nappi, JM.[2019]

In a study of 383 patients aged 80 and older with acute dyspnoea, BNP levels were significantly higher in those with cardiac dyspnoea compared to those with respiratory dyspnoea, indicating a potential link between BNP levels and heart failure.

Despite this association, BNP alone did not effectively distinguish between cardiac and respiratory causes of dyspnoea, suggesting limited clinical utility in diagnosing heart failure in very old patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Brain natriuretic peptide usefulness in very elderly dyspnoeic patients: the BED study. Plichart, M., Orvoën, G., Jourdain, P., et al.[2018]

Nesiritide, a synthetic version of brain natriuretic peptide (BNP), has been shown to effectively treat decompensated heart failure (HF) in over 2000 patients, demonstrating a rapid and sustained vasodilator effect without tachyphylaxis.

The safety profile of nesiritide is excellent, with dose-dependent hypotension as the main side effect, and it does not increase the risk of ventricular arrhythmias compared to placebo, making it a promising first-line treatment option for decompensated HF.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Brain natriuretic peptide (nesiritide) in the treatment of heart failure. Bettencourt, P.[2019]

Natriuretic peptides, particularly BNP and NT-proBNP, are essential for diagnosing heart failure and can also help assess the prognosis of patients, making them valuable biomarkers in clinical settings.

The introduction of angiotensin receptor neprilysin inhibitors (ARNIs) has increased the therapeutic relevance of natriuretic peptides, highlighting their potential role not just in diagnosis but also in the treatment of heart failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Natriuretic Peptides: Role in the Diagnosis and Management of Heart Failure: A Scientific Statement From the Heart Failure Association of the European Society of Cardiology, Heart Failure Society of America and Japanese Heart Failure Society. Tsutsui, H., Albert, NM., Coats, AJS., et al.[2023]

Natriuretic peptides (NPs), particularly ANP and BNP, are crucial for regulating blood pressure and fluid balance, and their levels increase in response to heart failure, serving as important biomarkers for diagnosis and prognosis.

The combination therapy of sacubitril/valsartan, which inhibits the enzyme that degrades NPs, has been clinically successful in reducing cardiovascular mortality and morbidity in heart failure patients, highlighting its efficacy compared to other therapeutic approaches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evolving Role of Natriuretic Peptides from Diagnostic Tool to Therapeutic Modality. Pagel-Langenickel, I.[2018]

Natriuretic peptides, particularly BNP and NT-proBNP, are essential for diagnosing heart failure and can also help assess the prognosis of patients, making them valuable biomarkers in clinical settings.

The introduction of angiotensin receptor-neprilysin inhibitors (ARNIs) has increased the therapeutic relevance of natriuretic peptides, highlighting their potential role not just in diagnosis but also in the treatment of heart failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Natriuretic peptides: role in the diagnosis and management of heart failure: a scientific statement from the Heart Failure Association of the European Society of Cardiology, Heart Failure Society of America and Japanese Heart Failure Society. Tsutsui, H., Albert, NM., Coats, AJS., et al.[2023]

Natriuretic peptides (NPs) like ANP and BNP play crucial roles in regulating heart failure by promoting natriuresis, diuresis, and vasodilation, acting as the body's natural antihypertensive system.

Current therapies, such as nesiritide and ARNI (LCZ696), have shown promising results in reducing morbidity and mortality in heart failure patients by enhancing NP activity and counteracting the effects of the renin-angiotensin-aldosterone system.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Synthesis, secretion, function, metabolism and application of natriuretic peptides in heart failure. Fu, S., Ping, P., Wang, F., et al.[2023]

References

1. Australia pubmed.ncbi.nlm.nih.gov

The role of b-type natriuretic peptide in heart failure management. [2015]

2. United States pubmed.ncbi.nlm.nih.gov

Novel protein therapeutics for systolic heart failure: chronic subcutaneous B-type natriuretic peptide. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

The role of B-type natriuretic peptide in heart failure. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Brain natriuretic peptide usefulness in very elderly dyspnoeic patients: the BED study. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

Brain natriuretic peptide (nesiritide) in the treatment of heart failure. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Natriuretic Peptides: Role in the Diagnosis and Management of Heart Failure: A Scientific Statement From the Heart Failure Association of the European Society of Cardiology, Heart Failure Society of America and Japanese Heart Failure Society. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Evolving Role of Natriuretic Peptides from Diagnostic Tool to Therapeutic Modality. [2018]

8. England pubmed.ncbi.nlm.nih.gov

Natriuretic peptides: role in the diagnosis and management of heart failure: a scientific statement from the Heart Failure Association of the European Society of Cardiology, Heart Failure Society of America and Japanese Heart Failure Society. [2023]

9. England pubmed.ncbi.nlm.nih.gov

Synthesis, secretion, function, metabolism and application of natriuretic peptides in heart failure. [2023]