DMD-Associated Dilated CardiomyopathyPhase 1MUSIC-DMDNCT06224660
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
A Phase 1 study testing SRD-001 in people with DMD-Associated Dilated Cardiomyopathy, run by Sardocor Corp. at 3 sites in United States.
Trial summary
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- DMD-Associated Dilated Cardiomyopathy
- What is being tested?
- SRD-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesUnited States
- Study dates
- 2 Oct 2024to Oct 2030
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of DMD with confirmatory genetic testing
- Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
- Left ventricular ejection fraction < 40%
- Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment
- Willing and able to provide informed consent
Who cannot join
- Abnormal blood pressure
- Non-DMD-related liver function test elevations
- Cystatin C ≥ 1.2 mg/L
- Thrombocytopenia
- Anemia
- Inadequate pulmonary function
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Heart Institute Neuromuscular Intake Line, 513-803-3000
Chet Villa, MD
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43215
Kelsey Craig, 614-722-2715, kelsey.craig@nationwidechildrens.org
Deipanjan Nandi, MD
- Recruiting
The University of Kansas Medical Center
Kansas City, Kansas, 66160
Jillian Bruenn, 913-588-9720, jfrick2@kumc.edu
Pradeep Mammen, MD
About this study
Official title: A Phase 1b, Open-Label, Controlled Trial Evaluating the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
Read the detailed description
This phase 1b, multi-center, non-randomized, open-label, ascending dose escalation, no-intervention-control trial will assess the safety and explore the efficacy of SRD-001 administered as a one-time antegrade epicardial coronary artery infusion for the treatment of participants with cardiomyopathy secondary to DMD. SRD-001 is an AAV1 vector expressing the transgene for SERCA2a. Twelve participants will be assigned to either active treatment with SRD-001 or no-intervention based upon their neutralizing antibody status. The objectives of the trial are (1) to evaluate the safety of a one-time intracoronary administration of SRD-001 in participants with cardiomyopathy due to DMD; and (2) to explore the impact of SRD-001 on heart and skeletal muscle function and quality of life. After screening to determine eligibility, participants will be sequentially assigned to low dose SRD-001, high dose SRD-001 or no-intervention. Participants assigned to active treatment with SRD-001 will under cardiac catheterization and angiography just prior to the intracoronary infusion of SRD-001 and spend overnight int he hospital for observation.
- Conditions
- DMD-Associated Dilated Cardiomyopathy
- Being tested
- SRD-001
- Medical subject headings
- Dmd-Associated Dilated Cardiomyopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRD-001Genetic
Also called AAV1/SSERCA2a
SRD-001 is an adeno-associated virus serotype 1 (AAV1) based gene therapy designed to deliver a copy of the gene encoding the human sarcoplasmic/endoplasmic reticulum Ca(2+) ATPase 2a (SERCA2a). It is administered as a one-time intracoronary infusion.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose | Experimental | SRD-001 Receives: Genetic: SRD-001 |
| High Dose | Experimental | SRD-001 Receives: Genetic: SRD-001 |
| Control | No intervention | No-Intervention Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of all-cause mortality
Death
Measured: From Day 1 to Week 52 and Week 104
Rate and severity of related treatment-emergent adverse events
Adverse events related to the investigational product or the administration procedure
Measured: From Day 1 to Week 52 and Week 104
Rate and severity of all treatment-emergent adverse events
Adverse events
Measured: From Day 1 to Week 52 and Week 104
Rate of cell-mediated immune reaction
Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)
Measured: From Day 1 to Week 52
Secondary outcomes (2)
Change, including normal/abnormal shifts, in 12-lead electrocardiogram (ECG)
Change in the heart's electrical activity
Measured: From Day 1 to Week 52 and Week 104
Change, including normal/abnormal shifts, in laboratory evaluations
Hematology, serum chemistries, urinalysis, cardiac enzymes and anti-AAV1 antibodies
Measured: From Day 1 to Week 52 and Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2024
- Study start2 Oct 2024
- Primary completionOct 2027 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DMD-Associated Dilated CardiomyopathyStudies 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 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
- NCT06224660
- Sponsor's protocol ID
- SRD-001-1004
- Acronym
- MUSIC-DMD
- Status last verified
- Feb 2025
- First submitted
- 11 Jan 2024
- Last updated
- 27 Feb 2025
- 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 27 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-Label, Controlled Trial Evaluating the Safety and Efficacy of SRD-001 (AAV1/SERCA2a) in Subjects With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06224660
- Acronym / sponsor ID
- MUSIC-DMD / SRD-001-1004
- Conditions
- DMD-Associated Dilated Cardiomyopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Sardocor Corp.Industry (pharma, biotech, devices)
- Study start
- 2 Oct 2024 (actual)
- Primary completion
- Oct 2027 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 25 Jan 2024 (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.
This research study is testing whether an experimental drug, called SRD-001, is safe and helps the weakened heart of patients with Duchenne muscular dystrophy (DMD) regain its ability to effectively pump blood to the rest of the body. SRD-001 is a form of gene therapy. The goal of SRD-001 gene therapy is to provide the heart muscle cells with extra copies of the SERCA2a gene so that they can produce more SERCA2a protein to help the heart muscle cells squeeze/contract better. Researchers will compare SRD-001 treated participants with no-treatment participants; all participants will continue to take their current heart medications. All participants will be followed very closely for 2 years and undergo cardiac magnetic resonance imaging of their heart at baseline, year 1 and year 2 along with assessment of upper limb function and lung function. After the 2 years of close follow-up, all participants will roll over into long-term follow-up where they will be called biannually for information on their current medical status.
Detailed description
This phase 1b, multi-center, non-randomized, open-label, ascending dose escalation, no-intervention-control trial will assess the safety and explore the efficacy of SRD-001 administered as a one-time antegrade epicardial coronary artery infusion for the treatment of participants with cardiomyopathy secondary to DMD. SRD-001 is an AAV1 vector expressing the transgene for SERCA2a. Twelve participants will be assigned to either active treatment with SRD-001 or no-intervention based upon their neutralizing antibody status. The objectives of the trial are (1) to evaluate the safety of a one-time intracoronary administration of SRD-001 in participants with cardiomyopathy due to DMD; and (2) to explore the impact of SRD-001 on heart and skeletal muscle function and quality of life. After screening to determine eligibility, participants will be sequentially assigned to low dose SRD-001, high dose SRD-001 or no-intervention. Participants assigned to active treatment with SRD-001 will under cardiac catheterization and angiography just prior to the intracoronary infusion of SRD-001 and spend overnight int he hospital for observation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose | Experimental | Genetic: SRD-001SRD-001 |
| High Dose | Experimental | Genetic: SRD-001SRD-001 |
| Control | No intervention | –No-Intervention Control |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of DMD with confirmatory genetic testing
- Cardiomyopathy with left ventricular scar in at least 3 of 16 segments
- Left ventricular ejection fraction < 40%
- Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment
- Willing and able to provide informed consent
Key exclusion criteria (6 listed)
- Abnormal blood pressure
- Non-DMD-related liver function test elevations
- Cystatin C ≥ 1.2 mg/L
- Thrombocytopenia
- Anemia
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-001Also: AAV1/SSERCA2aSRD-001 is an adeno-associated virus serotype 1 (AAV1) based gene therapy designed to deliver a copy of the gene encoding the human sarcoplasmic/endoplasmic reticulum Ca(2+) ATPase 2a (SERCA2a). It is administered as a one-time intracoronary infusion. | Genetic | High Dose, Low Dose |
Primary outcome
Rate of all-cause mortality
Time frame: From Day 1 to Week 52 and Week 104
Death
Rate and severity of related treatment-emergent adverse events
Time frame: From Day 1 to Week 52 and Week 104
Adverse events related to the investigational product or the administration procedure
Rate and severity of all treatment-emergent adverse events
Time frame: From Day 1 to Week 52 and Week 104
Adverse events
Rate of cell-mediated immune reaction
Time frame: From Day 1 to Week 52
Cell-mediated immune reaction as assessed by enzyme-linked immunosorbent spot (ELISpot)
2 secondary outcomes
Change, including normal/abnormal shifts, in 12-lead electrocardiogram (ECG)
Time frame: From Day 1 to Week 52 and Week 104
Change, including normal/abnormal shifts, in laboratory evaluations
Time frame: From Day 1 to Week 52 and Week 104
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 27 Feb 2025.
- 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.
3 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 SRD-001 in people with DMD-Associated Dilated Cardiomyopathy, run by Sardocor Corp. at 3 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 5 inclusion and 6 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 27 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 Corp. (+1-617-880-7616, info@sardocorcorp.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of all-cause mortality (time frame: From Day 1 to Week 52 and Week 104). The study lists 4 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 27 Feb 2025.