SRD-001 for Heart Failure

(MUSIC-HFrEF1 Trial)

Recruiting at 5 trial locations

Overseen By COO

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Sardocor Corp.

Must be taking: Guideline-directed therapy

Disqualifiers: Cardiomyopathy, Myocarditis, Cancer, others

Compensation: Varies

Number of Visits: Unknown

57 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called SRD-001 for individuals with heart failure. Researchers aim to determine if this treatment can improve heart function and blood flow by addressing a specific enzyme issue. Participants will receive either SRD-001 or a placebo (a harmless substance used for comparison) through a one-time heart infusion. Suitable candidates have heart failure with reduced ejection fraction (a measure of heart pumping efficiency) and are on standard heart failure medication. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that SRD-001 is likely to be safe for humans?

Research has shown that SRD-001, a gene therapy for heart failure, has promising safety results from earlier studies. This treatment involves a one-time infusion aimed at improving heart function by addressing specific enzyme problems. Reports from these studies indicate that patients generally tolerated SRD-001 well. While some side effects occurred, serious issues were rare. Overall, the data suggest that SRD-001 has a strong safety record so far, making it a promising option for those considering joining clinical trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about SRD-001 for heart failure because it offers a novel approach compared to traditional treatments like beta-blockers, ACE inhibitors, and diuretics. Unlike these standard treatments, which primarily manage symptoms and prevent the condition from worsening, SRD-001 is designed to be a one-time intracoronary infusion that delivers a high dose of viral vectors directly to the heart. This method aims to target the underlying causes of heart failure at the cellular level, potentially leading to more significant and longer-lasting improvements in heart function. The possibility of addressing the disease mechanism directly is what makes this treatment particularly promising.

What evidence suggests that SRD-001 might be an effective treatment for heart failure?

Research has shown that SRD-001, which participants in this trial may receive, might help people with heart failure by improving heart function. This treatment enhances the heart's pumping ability by addressing an issue with a specific enzyme. In earlier studies, SRD-001 demonstrated promising results for patients with a type of heart failure where the heart doesn't pump blood effectively. These findings suggest that SRD-001 could become a new treatment option for those with severe heart failure, addressing a significant unmet need. While more research is necessary, these initial results offer encouragement for those considering joining the trials. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• SRD-001

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Permanent pacing, especially left ventricular or biventricular pacing, can provide symptomatic relief for some patients with chronic heart failure (CHF), although its impact on survival rates is less clear.

The integration of implantable cardioverter-defibrillators (ICDs) with biventricular pacing may enhance treatment strategies for CHF patients by improving their functional status and reducing the risk of sudden cardiac death.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pacing for patients with congestive heart failure and dilated cardiomyopathy. Peters, RW., Gold, MR.[2019]

Citations

1. medera.bio medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/medera-completes-patient-dosing-in-phase-1/2a-music-hfref-trial-of-srd-001-gene-therapy-for-heart-failure

Medera Completes Patient Dosing MUSIC-HFrEF Trial The MUSIC-HFrEF trial is an open-label, uncontrolled study investigating SRD-001 across two cohorts with six patients dosed with SRD-001 in ...

2. medera.bio medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/2-clinical-trial-in-late-breaking-oral

Gene Therapy Data | Baltimore, MD - Medera “These encouraging results of SRD-001 in patients with HFrEF may offer alternative treatments to patients with severe heart failure where a large unmet need ...

3. biospace.com biospace.com/press-releases/medera-announces-first-patient-dosed-in-phase-2-portion-of-music-hfref-phase-1b-2-trial-evaluating-srd-001-gene-therapy-for-heart-failure-with-reduced-ejection-fraction

Medera Announces First Patient Dosed in Phase 2 Portion ... SRD-001 is an investigational gene therapy candidate that contains an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene ...

4. withpower.com withpower.com/trial/phase-2-heart-failure-9-2021-98a50

SRD-001 for Heart Failure (MUSIC-HFrEF1 Trial) While specific data on SRD-001 is not provided, similar treatments like carvedilol have shown improvements in heart function and survival in heart failure ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04703842

Study Details | NCT04703842 | Modulation of SERCA2a ... A Phase 1/2 Trial of the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Heart Failure With Reduced Ejection Fraction. Conditions. Congestive ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06061549

Study Details | NCT06061549 | Modulation of SERCA2a ... The goal of this clinical trial is to test an experimental gene therapy in participants with heart failure with preserved ejection fraction, also known as ...

7. cgtlive.com cgtlive.com/view/sardocor-heart-failure-gene-therapy-srd-001-garners-fast-track-designation-first-patients-dosed

Sardocor's Heart Failure Gene Therapy SRD-001 Garners ... Sardocor's SRD-001, an investigational adeno-associated virus (AAV) vector-based gene therapy being evaluated for the treatment of Heart failure with preserved ...