Heart Failure, DiastolicPhase 1MUSIC-HFpEFNCT06061549
Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction
A Phase 1 study testing AAV1/SERCA2a in people with Heart Failure, Diastolic and Heart Failure With Preserved Ejection Fraction, run by Sardocor Corp. at 2 sites in United States.
Trial summary
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 diastolic heart failure. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Heart Failure, Diastolic, Heart Failure With Preserved Ejection Fraction
- What is being tested?
- AAV1/SERCA2a
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Aug 2023to Aug 2029
- Phase
- Phase 1
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to provide informed consent
- Negative for anti-AAV1 neutralizing antibodies
- NYHA class II or III
- Left ventricular ejection fraction ≥ 50%
- Evidence of resting or exercise-induced left ventricle filling pressure
- On oral diuretic therapy
- Adequate birth control
Who cannot join
- NYHA class IV
- Heart failure requiring hospitalization in the past 3 months
- Manifested or provocable ischemic heart disease
- Atrial fibrillation
- History of congenital heart disease, restrictive or infiltrative cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm
- History of amyloidosis
Show all 12Show fewer
- Untreated left-sided valvular disease
- Severe COPD
- BMI > 50 kg/m^2
- Severe liver, kidney or hematologic dysfunction
- Cancer within the past 5 years
- Unstable concurrent conditions
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Not yet recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Therese Vallina, Therese.Vallina@UTSouthwestern.edu
- Recruiting
Duke University
Durham, North Carolina, 27705
Kay Doukellis, katheryn.doukellis@duke.edu
About this study
Official title: A Phase 1b, Pilot Trial Evaluating the Safety and Pharmacodynamic Effects of SRD-001 (AAV1-SERCA2a) in Subjects With Heart Failure With Preserved Ejection Fraction
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 diastolic heart failure. The main questions it aims to answer are:
- safety and tolerability of the gene therapy; and
- whether the gene therapy helps the heart ventricles relax during filling. Participants will undergo a one-time infusion of the gene therapy in the cardiac catheterization laboratory and then be followed for safety and effects on left-sided filling pressures while exercising. The first year will have multiple in-person visits followed by 4 years of biannual phone calls.
Read the detailed description
Eligible participants will undergo a one-time antegrade epicardial coronary artery infusion of gene therapy delivered to the 3 major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or bypass grafts unless contraindicated. Post-procedure, the subject will be monitored continuously for a minimum of 4 hours and then discharged to home that same day if medically appropriate. Participants will be followed for 52 weeks as part of the main trial. On Day 2 and Week 1, subjects will be contacted by phone for a general safety assessment, and if clinically indicated, an in-person evaluation and assessment will be performed as soon as possible. On Weeks 2, 4, 12, 24 and 52, subjects will undergo general safety assessments. Additionally, at Weeks 24 and 52, subjects will undergo outpatient core resting and exercise transthoracic echocardiography, right heart catheterization with hemodynamic monitoring and gas exchange measurements, 6-minute wall test, cardiac biomarker testing and completion the Kansas City Cardiomyopathy Questionnaire. Upon completion of the active observation period, participants will continue to be followed for 4 years with bi-annual, semi-structured telephone or in-person questionnaires that collect information on overall status and other comorbid conditions of interest in gene therapy trials.
- Being tested
- AAV1/SERCA2a
- Medical subject headings
- Heart Failure, Diastolic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV1/SERCA2aBiological
3E13 viral genomes fixed dose
| Study group | Role | What they receive |
|---|---|---|
| SRD-001 Gene Therapy | Experimental | AAV1/SERCA2a 3E13 vg Receives: Biological: AAV1/SERCA2a |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in pulmonary capillary wedge pressure (PCWP)
PCWP assessed by right heart catheterization; change in mmHg from baseline
Measured: Week 24 and Week 52
Secondary outcomes (11)
Change in PCWP at 20W exercise
PCWP assessed by right heart catheterization; change in mmHg from baseline
Measured: Week 24 and Week 52
Change in oxygen uptake (VO2) during exercise
VO2 assessed by bicycle exercise; change in mL/min from baseline
Measured: Week 24 and Week 52
Change in left ventricular (LV) relaxation
Assessed by transesophageal echocardiography (ultrasound) using 2 methods: LV global peak early diastolic strain rate (1/s) and LV tau
Measured: Week 24 and Week 52
Change in NT-proBNP
Laboratory measurement (picograms per mL) in blood; high levels indicate the heart is working too hard to pump blood; decrease over time would indicate an improvement in heart failure condition
Measured: Week 24 and Week 52
Change in left atrial end systolic volume
Assessed by transesophageal echocardiography (ultrasound); change in mL/m2 from baseline
Measured: Week 24 and Week 52
Change in left atrial reservoir strain
Assessed by transesophageal echocardiography (ultrasound); change in %from baseline
Measured: Week 24 and Week 52
Change in left atrial contractile strain
Assessed by transesophageal echocardiography (ultrasound); change in % from baseline
Measured: Week 24 and Week 52
Change in 6 Minute Walk Distance
Distance walked in 6 minutes, measured in meters; the longer distance walked, the better medical condition
Measured: Week 24 and Week 52
Change in 6 Minute Walk Test Borg scale
Self-rated shortness of breath score with scale from 0 to 10, ranging from none to very, very severe
Measured: Week 24 and Week 52
Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) score
Measures symptoms, physical and social limitations, and quality of life in patients with heart failure; 23-item self-administered questionnaire; score of 0 to 100; 0-24, very poor to poor; 25-49, poor to fair; 50 to 74, faire to good; and 75 to 100, good to excellent
Measured: Week 24 and Week 52
Change in New York Heart Association class
Classification of heart failure based on severity of symptoms; 4 classes, I, II, III or IV where class I is having no symptoms doing ordinary physical activity; class II is having fatigue, shortness of breath, palpitations or angina with ordinary physical activity; class II is being comfortable only at rest; and class IV is having symptoms even at rest
Measured: Week 24 and Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2023
- Study start24 Aug 2023
- Primary completionAug 2025 (estimated)
- Study completionAug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Heart Failure, DiastolicStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 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
- NCT06061549
- Sponsor's protocol ID
- SRD-001-1003
- Acronym
- MUSIC-HFpEF
- Status last verified
- Sep 2023
- First submitted
- 30 Jul 2023
- Last updated
- 29 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Fudim M, Sarma S, MacNamara JP, Ascheim DD, Kittipibul V, Doukellis K, Dhimitri A, Fitzhugh R, Kamat N, Costa KD, Rudy J, Guerrero J, Chan C, Li RA, Bavry AA, Patel MR, Grodin JL, Levine BD, Hajjar RJ. Rationale and design of MUSIC-HFpEF: a phase 1b, pilot trial evaluating the safety and pharmacodynamic effects of AAV1.SERCA2A in heart failure with preserved ejection fraction. Heart Fail Rev. 2026 Jan 17;31(1):19. doi: 10.1007/s10741-025-10589-w.
PubMed 41546750
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Pilot Trial Evaluating the Safety and Pharmacodynamic Effects of SRD-001 (AAV1-SERCA2a) in Subjects With Heart Failure With Preserved Ejection Fraction
- ClinicalTrials.gov ID
- NCT06061549
- Acronym / sponsor ID
- MUSIC-HFpEF / SRD-001-1003
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Sardocor Corp.Industry (pharma, biotech, devices)
- Study start
- 24 Aug 2023 (actual)
- Primary completion
- Aug 2025 (estimated)
- Study completion
- Aug 2029 (estimated)
- First posted
- 29 Sep 2023 (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.
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 diastolic heart failure. The main questions it aims to answer are:
- safety and tolerability of the gene therapy; and
- whether the gene therapy helps the heart ventricles relax during filling. Participants will undergo a one-time infusion of the gene therapy in the cardiac catheterization laboratory and then be followed for safety and effects on left-sided filling pressures while exercising. The first year will have multiple in-person visits followed by 4 years of biannual phone calls.
Detailed description
Eligible participants will undergo a one-time antegrade epicardial coronary artery infusion of gene therapy delivered to the 3 major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or bypass grafts unless contraindicated. Post-procedure, the subject will be monitored continuously for a minimum of 4 hours and then discharged to home that same day if medically appropriate. Participants will be followed for 52 weeks as part of the main trial. On Day 2 and Week 1, subjects will be contacted by phone for a general safety assessment, and if clinically indicated, an in-person evaluation and assessment will be performed as soon as possible. On Weeks 2, 4, 12, 24 and 52, subjects will undergo general safety assessments. Additionally, at Weeks 24 and 52, subjects will undergo outpatient core resting and exercise transthoracic echocardiography, right heart catheterization with hemodynamic monitoring and gas exchange measurements, 6-minute wall test, cardiac biomarker testing and completion the Kansas City Cardiomyopathy Questionnaire. Upon completion of the active observation period, participants will continue to be followed for 4 years with bi-annual, semi-structured telephone or in-person questionnaires that collect information on overall status and other comorbid conditions of interest in gene therapy trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SRD-001 Gene Therapy | Experimental | Biological: AAV1/SERCA2aAAV1/SERCA2a 3E13 vg |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Willing and able to provide informed consent
- Negative for anti-AAV1 neutralizing antibodies
- NYHA class II or III
- Left ventricular ejection fraction ≥ 50%
- Evidence of resting or exercise-induced left ventricle filling pressure
Key exclusion criteria (12 listed)
- NYHA class IV
- Heart failure requiring hospitalization in the past 3 months
- Manifested or provocable ischemic heart disease
- Atrial fibrillation
- History of congenital heart disease, restrictive or infiltrative cardiomyopathy, hypertrophic cardiomyopathy, acute myocarditis, pericardial disease, uncorrected thyroid disease or discrete left ventricular (LV) aneurysm
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 |
|---|---|---|
| AAV1/SERCA2a3E13 viral genomes fixed dose | Biological | SRD-001 Gene Therapy |
Primary outcome
Change in pulmonary capillary wedge pressure (PCWP)
Time frame: Week 24 and Week 52
PCWP assessed by right heart catheterization; change in mmHg from baseline
11 secondary outcomes
Change in PCWP at 20W exercise
Time frame: Week 24 and Week 52
Change in oxygen uptake (VO2) during exercise
Time frame: Week 24 and Week 52
Change in left ventricular (LV) relaxation
Time frame: Week 24 and Week 52
Change in NT-proBNP
Time frame: Week 24 and Week 52
Change in left atrial end systolic volume
Time frame: Week 24 and Week 52
Change in left atrial reservoir strain
Time frame: Week 24 and Week 52
Change in left atrial contractile strain
Time frame: Week 24 and Week 52
Change in 6 Minute Walk Distance
Time frame: Week 24 and Week 52
Change in 6 Minute Walk Test Borg scale
Time frame: Week 24 and Week 52
Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) score
Time frame: Week 24 and Week 52
Change in New York Heart Association class
Time frame: Week 24 and Week 52
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 29 Sep 2023.
- Derived from this study: PubMed 41546750Fudim M, Sarma S, MacNamara JP, Ascheim DD, Kittipibul V, Doukellis K, Dhimitri A, Fitzhugh R, Kamat N, Costa KD, Rudy J, Guerrero J, Chan C, Li RA, Bavry AA, Patel MR, Grodin JL, Levine BD, Hajjar RJ. Rationale and design of MUSIC-HFpEF: a phase 1b, pilot trial evaluating the safety and pharmacodynamic effects of AAV1.SERCA2A in heart failure with preserved ejection fraction. Heart Fail Rev. 2026 Jan 17;31(1):19. doi: 10.1007/s10741-025-10589-w.
- Individual participant dataSharing planned: No
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV1/SERCA2a in people with Heart Failure, Diastolic and Heart Failure With Preserved Ejection Fraction, run by Sardocor Corp. at 2 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?
The sponsor lists 7 inclusion and 12 exclusion criteria. 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 29 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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 Sardocor (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 in pulmonary capillary wedge pressure (PCWP) (time frame: Week 24 and Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06061549, which also lists 1 linked publication.
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 29 Sep 2023.