NCT04703842 · Sardocor Corp.
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction
(MUSIC-HFrEF1)
What this study is about
It is believed that targeted SERCA2a enzyme replacement in HFrEF patients will correct defective intracellular Ca2+ hemostasis, resulting in improved cardiac contractile function and energetics which will, in turn, translate to improved clinical outcomes.
View original scientific description
It is believed that targeted SERCA2a enzyme replacement in HFrEF patients will correct defective intracellular Ca2+ hemostasis, resulting in improved cardiac contractile function and energetics which will, in turn, translate to improved clinical outcomes. Additionally, it is hypothesized that correcting SERCA2a dysfunction will also improve coronary blood flow through correction of the impaired endothelium-dependent nitric oxide-mediated vasodilatation observed in heart failure.
Interventions
BIOLOGICAL
SRD-001
AAV1/SERCA2a
DRUG
Placebo
SRD-001 matching placebo
Primary outcome measures
Change from baseline in symptomatic parameters
Time frame: Baseline to Month 6 and Month 12
New York Heart Association classification (I, II, III or IV)
Change from baseline in symptomatic parameters
Time frame: Baseline to Month 6 and Month 12
Quality of life as assessed by Kansas City Cardiomyopathy Questionnaire: 0-24, very poor to poor; 25-49, poor to fair; 50-74, fair to good; and 75-100, good to excellent
Change from baseline in physical parameter
Time frame: Baseline to Month 6 and Month 12
Distance walked during the 6MWT
Change from baseline in LV function/remodeling
Time frame: Baseline to Month 6 and Month 12
Left ventricular end systolic volume (LVESV) as assessed by echocardiography
Rate of recurrent events
Time frame: Baseline to Month 6 and Month 12
HF-related hospitalization, ambulatory worsening heart failure, all-cause death, MCSD and transplant
Rate of adverse events
Time frame: 6 and 12 months
Treatment-emergent adverse events
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Chronic ischemic or non-ischemic cardiomyopathy
- NYHA class III/IV
- Guideline-directed medical therapy for heart failure; ICD Main
Exclusion criteria
- Restrictive cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm
- Prior heart transplantation, left ventricular reduction surgery (LVRS), cardiomyoplasty, passive restraint device (e.g., CorCap™ Cardiac Support Device), mechanical circulatory support device (MCSD) or cardiac shunt
- Likely to receive cardiac resynchronization therapy, cardiomyoplasty, LVRS, conventional revascularization procedure or valvular repair in the 6 months following treatment
- Likely need for an immediate heart transplant or MCSD implant due to hemodynamic instability
- Inadequate hepatic and renal function
- Diagnosis of, or treatment for, any cancer within the last 5 yea
Where
- San Diego, California
- San Francisco, California
- St Louis, Missouri
- Dallas, Texas
- Seattle, Washington
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 26, 2024 · Source of record for eligibility and locations