What side effects are associated with this type of intervention?

Common treatments for heart failure include medications like ACE inhibitors, beta-blockers, diuretics, and newer agents such as angiotensin receptor-neprilysin inhibitors (ARNIs). Gene therapy, which is being studied for its potential to improve ventricular relaxation, is an emerging treatment. Known side effects of these treatments can vary: ACE inhibitors may cause cough and hyperkalemia; beta-blockers can lead to fatigue and bradycardia; diuretics might result in electrolyte imbalances; and ARNIs can cause hypotension and renal impairment. Gene therapy's side effects are still being studied, but potential risks include immune reactions and off-target genetic effects.

What prior FDA approvals exist?

The FDA has approved several treatments for heart failure, including medications like ACE inhibitors, beta-blockers, and diuretics, which manage symptoms and improve heart function. More recently, gene therapy approaches, such as the experimental therapy targeting SERCA2a, aim to improve ventricular relaxation by modifying genetic material. This therapy involves a one-time infusion of a gene therapy vector in the cardiac catheterization laboratory, followed by monitoring for safety and efficacy. While traditional treatments focus on symptom management, gene therapy represents a novel approach by addressing the underlying genetic causes of heart failure.

What other types of research exist?

Promising novel alternatives to gene therapy for heart failure patients include: 1) Sodium-glucose co-transporter-2 (SGLT2) inhibitors, which have shown benefits in heart failure with preserved ejection fraction (HFpEF) by improving diastolic function and reducing hospitalization rates. 2) Soluble guanylate cyclase (sGC) stimulators, which enhance nitric oxide signaling and improve myocardial relaxation and vascular function. 3) Myosin modulators, such as omecamtiv mecarbil, which improve cardiac contractility and efficiency. These alternatives are currently being evaluated in clinical trials and may offer effective treatment options in 2024.

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Gene Therapy

Gene Therapy for Diastolic Heart Failure (MUSIC-HFpEF Trial)

Phase 1

Recruiting

Research Sponsored by Sardocor Corp.

Eligibility Criteria Checklist

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

Must have

NYHA class II or III

On oral diuretic therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 24 and week 52

Awards & highlights

MUSIC-HFpEF Trial Summary

This trial tests a gene therapy to help people with diastolic heart failure. Participants get a one-time infusion and follow-up for safety and changes in heart function. Long-term follow-up includes yearly in-person visits and biannual phone calls.

Who is the study for?

This trial is for adults with heart failure who still have a good amount of pumping function (ejection fraction ≥ 50%) and symptoms that are not the most severe. They should be able to exercise somewhat, take diuretics, and use birth control if needed. People with severe lung disease, extreme obesity, unstable conditions, major organ dysfunction or specific heart diseases like amyloidosis can't join.Check my eligibility

What is being tested?

The study tests a one-time gene therapy infusion called AAV1/SERCA2a to see if it's safe and can help the heart fill better during relaxation. Participants will receive this treatment in a special lab setting and then be monitored closely for one year with follow-ups over phone calls for four more years.See study design

What are the potential side effects?

Potential side effects may include reactions at the infusion site or allergic responses due to the body's reaction to the new gene material. Since it involves genetic therapy, there might also be unforeseen risks related to altering genes within heart muscle cells.

MUSIC-HFpEF Trial Eligibility Criteria

Inclusion Criteria

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

Select...

My heart condition mildly or moderately affects my daily activities.

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I am currently taking water pills.

MUSIC-HFpEF Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 24 and week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 24 and week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 24 and week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in pulmonary capillary wedge pressure (PCWP)

Secondary outcome measures

Change in 6 Minute Walk Distance

Change in 6 Minute Walk Test Borg scale

Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) score

+8 more

MUSIC-HFpEF Trial Design

1Treatment groups

Experimental Treatment

Group I: SRD-001 Gene TherapyExperimental Treatment1 Intervention

AAV1/SERCA2a 3E13 vg

0 / 2Eligibility 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

Gene therapy for heart failure, such as the one being studied to improve ventricular relaxation, works by modifying genetic material to enhance the function of proteins involved in cardiac muscle contraction and relaxation. This can help the heart fill more effectively during diastole, improving overall cardiac function. Other common treatments include inotropes, which increase the force of heart contractions, and vasodilators, which reduce the resistance the heart must pump against, thereby improving blood flow and reducing the heart's workload. Mechanical support devices, like ventricular assist devices, can take over some of the heart's pumping function, providing relief to the failing heart. These treatments are crucial for heart failure patients as they aim to improve symptoms, enhance quality of life, and reduce the risk of hospitalization and mortality.

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Who is running the clinical trial?

Sardocor Corp.Lead Sponsor

2 Previous Clinical Trials

69 Total Patients Enrolled

1 Trials studying Heart Failure

57 Patients Enrolled for Heart Failure

~6 spots leftby Aug 2025

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