Congestive Heart FailurePhase 1/2MUSIC-HFrEF1NCT04703842
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction
A Phase 1/2 study testing SRD-001 in people with Congestive Heart Failure and Heart Failure, Systolic, run by Sardocor Corp. at 5 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Congestive Heart Failure, Heart Failure, Systolic, Heart Failure
- What is being tested?
- SRD-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 57 participants
- Where
- 5 sitesUnited States
- Study dates
- 23 Sep 2021to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Main Inclusion Criteria:
- Chronic ischemic or non-ischemic cardiomyopathy
- NYHA class III/IV
- LVEF ≤35%
- 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 years except for basal cell carcinoma or carcinomas in situ where surgical excision was considered curative
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Therese Villina, MPHI, BSN, 214-645-7083, Therese.Vallina@utsouthwestern.edu
Justin Grodin, MD
- Recruiting
San Diego Cardiac Center
San Diego, California, 92123
Emma Burkhalter, RN, 858-244-6890, eburkhalter@sdcardiac.com
Brian Jaski, MD
- Recruiting
University of California, San Francisco
San Francisco, California, 94143
Kaye Reambonanza, 415-514-6147, Kaye.Reambonanza@ucsf.edu
Richard Cheng, MD
- Recruiting
University of Washington Medicine
Seattle, Washington, 98195
Katherine Nassans, 206-543-6850, Knassans@uw.edu
Kenta Nakamura, MD
- Recruiting
Washington University in Saint Louis
St Louis, Missouri, 63110
Annie Dirks, RN, BSN, 314-454-8711, aplatts@wustl.edu
Gregory Ewald, MD
About this study
Official title: A Phase 1/2 Trial of the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Heart Failure With Reduced Ejection Fraction
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.
Read the detailed description
MUSIC-HFrEF1 is an interventional study of SRD-001, an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), in anti-AAV1 neutralizing antibody (NAb) negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association (NYHA) class III/IV symptoms of heart failure with reduced ejection fraction (HFrEF).
The Phase 1 trial is an open-label, uncontrolled study investigating two doses of SRD-001 at 3E13 and 4.5E13 viral genomes (vg) in 3-4 participants at each dose level.
The Phase 2 trial is a randomized, double-blind, placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 1:1 ratio and a total sample size of N=50.
- Conditions
- Congestive Heart Failure, Heart Failure, Systolic, Heart Failure, HFrEF - Heart Failure With Reduced Ejection Fraction
- Being tested
- SRD-001, Placebo
- Medical subject headings
- Heart Failure, Heart Failure, Systolic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRD-001Biological
AAV1/SERCA2a
PlaceboDrug
SRD-001 matching placebo
| Study group | Role | What they receive |
|---|---|---|
| SRD-001 | Experimental | 3E13 or 4.5E13 vg; one-time intracoronary infusion Receives: Biological: SRD-001 |
| Placebo | Placebo comparator | One-time intracoronary infusion Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in symptomatic parameters
New York Heart Association classification (I, II, III or IV)
Measured: Baseline to Month 6 and Month 12
Change from baseline in symptomatic parameters
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
Measured: Baseline to Month 6 and Month 12
Change from baseline in physical parameter
Distance walked during the 6MWT
Measured: Baseline to Month 6 and Month 12
Change from baseline in LV function/remodeling
Left ventricular end systolic volume (LVESV) as assessed by echocardiography
Measured: Baseline to Month 6 and Month 12
Rate of recurrent events
HF-related hospitalization, ambulatory worsening heart failure, all-cause death, MCSD and transplant
Measured: Baseline to Month 6 and Month 12
Rate of adverse events
Treatment-emergent adverse events
Measured: 6 and 12 months
Secondary outcomes (6)
Proportion of subjects who complete the trial
Those who complete the trial vs withdraw consent, are lost to follow-up, or withdrawn due to an AE or other reason
Measured: 12 months
Concomitant medication use
Changes in heart failure related medications
Measured: 6 and 12 months
Incidence of abnormal laboratory test results
Hematologic, serum chemistries, NT-proBNP and troponin
Measured: Baseline to Month 6 and Month 12
Incidence of abnormal ECG results
New arrhythmias
Measured: Baseline to Month 6 and Month 12
Incidence of abnormal physical examination findings
Change from baseline in physical examination findings
Measured: Baseline to Month 6 and Month 12
Incidence of abnormal findings from interrogation of implantable cardioverter defibrillator
New arrhythmias
Measured: Baseline to Month 6 and Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2021
- Study start23 Sep 2021
- Primary completion31 Dec 2025 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congestive Heart FailureStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 57 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sardocor Corp.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04703842
- Sponsor's protocol ID
- SRD-001-1001
- Acronym
- MUSIC-HFrEF1
- Status last verified
- Mar 2024
- First submitted
- 7 Jan 2021
- Last updated
- 26 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Trial of the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Heart Failure With Reduced Ejection Fraction
- ClinicalTrials.gov ID
- NCT04703842
- Acronym / sponsor ID
- MUSIC-HFrEF1 / SRD-001-1001
- Conditions
- Congestive Heart Failure, Heart Failure, Systolic, Heart Failure, HFrEF - Heart Failure With Reduced Ejection Fraction
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 57 participants (estimated)
- Lead sponsor
- Sardocor Corp.Industry (pharma, biotech, devices)
- Study start
- 23 Sep 2021 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 11 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
MUSIC-HFrEF1 is an interventional study of SRD-001, an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), in anti-AAV1 neutralizing antibody (NAb) negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association (NYHA) class III/IV symptoms of heart failure with reduced ejection fraction (HFrEF).
The Phase 1 trial is an open-label, uncontrolled study investigating two doses of SRD-001 at 3E13 and 4.5E13 viral genomes (vg) in 3-4 participants at each dose level.
The Phase 2 trial is a randomized, double-blind, placebo-controlled trial with prospective assignment to a single dose of either SRD-001 or placebo in a 1:1 ratio and a total sample size of N=50.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SRD-001 | Experimental | Biological: SRD-0013E13 or 4.5E13 vg; one-time intracoronary infusion |
| Placebo | Placebo comparator | Drug: PlaceboOne-time intracoronary infusion |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SRD-001AAV1/SERCA2a | Biological | SRD-001 |
| PlaceboSRD-001 matching placebo | Drug | Placebo |
Primary outcome
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
6 secondary outcomes
Proportion of subjects who complete the trial
Time frame: 12 months
Concomitant medication use
Time frame: 6 and 12 months
Incidence of abnormal laboratory test results
Time frame: Baseline to Month 6 and Month 12
Incidence of abnormal ECG results
Time frame: Baseline to Month 6 and Month 12
Incidence of abnormal physical examination findings
Time frame: Baseline to Month 6 and Month 12
Incidence of abnormal findings from interrogation of implantable cardioverter defibrillator
Time frame: Baseline to Month 6 and Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2024.
- Individual participant dataSharing planned: No
Related trials for Congestive Heart Failure
RecruitingPhase 4
The Effects of Ferric Derisomaltose in Patients With Acute Heart Failure and Iron Deficiency on Exercise Capacity and Quality of Life(COREVIVE-HFrEF)
Heart Failure With Reduced Ejection Fraction, Iron Deficiency
Not yet recruitingPhase 2
A Study of Bimagrumab in Participants With HFpEF
Heart Failure, Diastolic, Diastolic Heart Failure
Recruiting
Evaluation of a French Hospital Discharge Therapeutic Education Program by Nurses for Heart Failure Patient (EduSor)
Heart Failure
Active, not recruiting
Assessment of Combined CCM and ICD Device in HFrEF
Heart Failure, Heart Failure With Reduced Ejection Fraction
Not yet recruiting
RELIEVE-HFrEF TRIAL: REducing Lung congestIon Symptoms Using the v-wavE Shunt in adVancEd Heart Failure With Reduced EF
Heart Failure and Reduced Ejection Fraction, Heart Failure Chronic
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SRD-001 in people with Congestive Heart Failure and Heart Failure, Systolic, run by Sardocor Corp. at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact COO (858-752-2941, info@sardocorcorp.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in symptomatic parameters (time frame: Baseline to Month 6 and Month 12). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2024.