SRD-001 for Heart Failure

(MUSIC-HFrEF1 Trial)

Recruiting at4 trial locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Sardocor Corp.

Must be taking: Guideline-directed therapy

Disqualifiers: Cardiomyopathy, Myocarditis, Cancer, others

Trial Summary

What is the purpose of this trial?

This trial uses a harmless virus to deliver a gene that helps heart cells produce a beneficial protein. It targets patients with severe heart failure who don't respond well to other treatments. The treatment works by improving calcium handling in heart cells, which enhances heart function and blood flow. Gene therapy has recently emerged as a powerful tool offering the promise of a new paradigm for alleviating heart failure.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it mentions that participants should be on guideline-directed medical therapy for heart failure. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the treatment SRD-001 for heart failure?

The research highlights that effective treatments for heart failure should improve survival, reduce hospitalizations, and enhance functional capacity. While specific data on SRD-001 is not provided, similar treatments like carvedilol have shown improvements in heart function and survival in heart failure patients, suggesting potential benefits for SRD-001. ^{1 2 3 4 5}

Research Team

Eligibility Criteria

This trial is for adults with heart failure and reduced ejection fraction (LVEF ≤35%). Participants must be in NYHA class III/IV, have an ICD implanted, and be on the best possible heart failure medications. People with poor liver or kidney function, recent cancer treatments (except certain skin cancers), likely need for a heart transplant or mechanical support soon, or specific other heart conditions are not eligible.

Inclusion Criteria

I am on the best possible treatment for heart failure and have an ICD.

I have a long-term heart muscle condition.

I am following doctor-recommended treatments for heart failure.

Exclusion Criteria

I do not have specific heart or thyroid conditions.

My liver and kidney functions are not normal.

I have had heart surgery or use a heart support device.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Treatment

Open-label, uncontrolled study investigating two doses of SRD-001 at 3E13 and 4.5E13 viral genomes in 3-4 participants at each dose level

6 months

One-time intracoronary infusion

Phase 2 Treatment

Randomized, double-blind, placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 1:1 ratio

6 months

One-time intracoronary infusion

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• SRD-001 (Enzyme Replacement)

Trial Overview The study tests whether SRD-001 can improve heart muscle function by correcting calcium handling inside cells. It's compared to a placebo. The idea is that fixing SERCA2a enzyme levels could help hearts pump better and improve blood flow through arteries by aiding vessel relaxation.

Participant Groups

Group I: SRD-001 Experimental Treatment 1 Intervention

3E13 or 4.5E13 vg; one-time intracoronary infusion

Group II: Placebo Placebo Group 1 Intervention

One-time intracoronary infusion

Who Is Running the Clinical Trial?

Sardocor Corp.

Lead Sponsor

Trials

3

Recruited

80+

Covance

Industry Sponsor

Trials

124

Recruited

13,300+

Dr. Paul Kirchgraber

Covance

Chief Executive Officer since 2019

MD

Dr. Robert Dow

Covance

Chief Medical Officer since 2020

MD

Findings from Research

New therapeutic agents for heart failure can gain approval if they show improvements in survival rates, reduce hospitalizations, or safely enhance patients' functional capacity.

Recent regulatory practices emphasize the use of composite endpoints in clinical trials to efficiently assess the efficacy and safety of new heart failure treatments, including biologicals and cell therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Trial Design, Endpoints, and Regulatory Requirements. Rosano, GMC.[2017]

Device-based therapies, such as implantable defibrillators, are crucial for improving outcomes in heart failure patients, especially those who have experienced life-threatening arrhythmias like ventricular fibrillation.

Cardiac resynchronization therapy (CRT) shows promise in alleviating heart failure symptoms and is currently being evaluated in large trials (CARE-HF and COMPANION) for its impact on patient morbidity and mortality, highlighting the need for careful patient selection and trial integrity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcome studies with device therapy in patients with heart failure. Cleland, JG., Thackray, S., Goodge, L., et al.[2019]

Effective drug and device therapies for heart failure (HF) are essential to reduce the high rates of morbidity and mortality associated with the condition, highlighting the importance of choosing appropriate clinical trial endpoints.

Incorporating patient-reported outcomes and functional capacity measures alongside traditional endpoints like mortality and hospitalizations is crucial for understanding the full impact of treatments on HF patients' quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Endpoints in Heart Failure Drug Development. Hussain, A., Misra, A., Bozkurt, B.[2022]

Carvedilol administration in heart failure patients enhances hemodynamic function, baroreflex sensitivity, and heart rate variability, which are crucial for heart health.

These improvements are associated with better survival rates in patients with heart failure, highlighting carvedilol's efficacy as a treatment option.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of carvedilol on heart rate and blood pressure variability in subjects with chronic heart failure. Piccirillo, G., Luparini, RL., Celli, V., et al.[2022]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Clinical Trial Design, Endpoints, and Regulatory Requirements. [2017]

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

Outcome studies with device therapy in patients with heart failure. [2019]

3. England pubmed.ncbi.nlm.nih.gov

Endpoints in Heart Failure Drug Development. [2022]

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

Effects of carvedilol on heart rate and blood pressure variability in subjects with chronic heart failure. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Pacing for patients with congestive heart failure and dilated cardiomyopathy. [2019]