Duchenne Muscular DystrophyPhase 2HOPENCT02485938
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
A Phase 2 study testing Allogeneic Cardiosphere-Derived Cells (CAP-1002) in people with Duchenne Muscular Dystrophy and Cardiomyopathy, run by Capricor Inc. at 3 sites in United States.
Trial summary
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Cardiomyopathy
- What is being tested?
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 3 sitesUnited States
- Study dates
- 7 Jan 2016to 14 Sep 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male participants 18 years of age or older must be able to provide informed consent and follow up with protocol procedures. Male participants at least 12 years of age but younger than 18 years of age must be able to provide assent with parent or guardian providing permission for study participation. Only male participants will be randomized into this study.
- Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.
- Cardiomyopathy with left ventricular scar by late gadolinium enhancement (LGE) in at least 4 segments as assessed by contrast-enhanced MRI and EF >35% at the time of screening.
- Use of evidence based medical-therapy in accordance with the "DMD Care Considerations Working Group" guidelines for the management of DMD, for at least three months prior to signing the consent form (or, providing assent) or documented contraindication or intolerance or patient preference.
- Participants must be taking systemic glucocorticoids for at least six months prior to screening.
- Participants must be 12 years of age or older at time of screening
- Participants must be appropriate candidates for cardiac catheterization and intracoronary infusion of CAP-1002, in the judgement of the site's interventional cardiologist.
Who cannot join
- Therapy with intravenous inotropic or vasoactive medications at the time of screening.
- Inability to undergo cardiac catheterization and/or MRI without general anesthesia.
- Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling.
- Planned or likely major surgery in the next 12 months after planned randomization.
- Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD.
- Contraindication to cardiac MRI.
Show all 27Show fewer
- Known hypersensitivity to contrast agents.
- Estimated glomerular filtration rate (GFR) <60 mL/min, as calculated by the CKD-EPI cystatin C equation (Inker, Schmid et al. 2012).
- Active infection not responsive to treatment.
- Active systemic allergic reaction(s), connective tissue disease or autoimmune disorder(s).
- History of cardiac tumor or cardiac tumor demonstrated on screening MRI.
- History of previous stem cell therapy.
- History of use of medications listed in Appendix 3 within 3 months prior to signing the Inform Consent Form / Assent through completion of the study infusion.
- Known moderate-to-severe aortic stenosis/insufficiency or severe mitral stenosis/regurgitation.
- Current active alcohol or drug abuse.
- Known history of Human Immunodeficiency Virus (HIV) infection.
- Known history of chronic viral hepatitis.
- Abnormal liver function (alanine aminotransferase (ALT)/aspartate aminotransferase (AST) >10 times the upper reference range) and/or abnormal hematology (hematocrit <25%, White Blood Cells <3000 μl, platelets <100,000 μl) studies without a reversible, identifiable cause.
- Known hypersensitivity to bovine products.
- Known hypersensitivity to dimethyl sulfoxide (DMSO).
- Uncontrolled diabetes (HbA1c >9.0).
- Inability to comply with protocol-related procedures, including required study visits.
- Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study.
- Currently receiving investigational treatment on another clinical study or expanded access protocol, including any of the following:
- Received investigational intervention within 30 days prior to randomization
- Treatment and/or an incomplete follow-up to treatment with any investigational cell based therapy within 6 months prior to randomization
- Active participation in other research therapy for cardiovascular repair/regeneration
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Florida
Gainesville, Florida, 32611
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
Official title: A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.
Read the detailed description
Approximately 24, and not more than 30, participants will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2.
The first 6-8 randomized participants will comprise Group 1, and will include a minimum of 3 participants completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused participants and for at least 6 participants overall.
Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 participants. Screening and randomization will continue until at total of 12 participants are infused with CAP 1002 or 30 participants are randomized into the study, whichever comes first.
All participants assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior).
Participants randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the participant on an ongoing basis, and will receive no infusion.
Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible participants randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible participants randomized to the usual care arm will occur 7 days after the date of randomization. All randomized participants will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.
- Conditions
- Duchenne Muscular Dystrophy, Cardiomyopathy
- Being tested
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Keywords
- Duchenne Muscular Dystrophy, Cardiomyopathy
- Medical subject headings
- Muscular Dystrophy, Duchenne, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Cardiosphere-Derived Cells (CAP-1002)Drug
Also called CAP-1002
Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002)
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Experimental | CAP-1002 is an investigational product consisting of allogeneic cardiosphere-derived cells (CDCs). All subjects assigned to the active treatment arm will receive an intended total dose of 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). If any of the three coronary arteries are deemed by the infusing Investigator to supply less than 30% of the left ventricular myocardium, the infusing Investigator may choose to infuse only 12.5M cells into that coronary artery or arteries. Therefore the full dose of CAP-1002 delivered may range from 50M cells to 75M cells provided that all three arteries are infused. Receives: Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002) |
| Usual Care | No intervention | Subjects randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the subject on an ongoing basis, and will receive no infusion. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing Any of the Adjudicated Events
Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations).
Measured: Within 72 hours post-infusion
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
Measured: Up to Month 12 post-infusion
Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12
Clinical chemistry parameters assessed were chloride, potassium and sodium.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12
Clinical chemistry parameter assessed was albumin.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12
Clinical chemistry parameter assessed was glucose.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12
Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12
Hematological parameter assessed was hemoglobin.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12
Hematological parameter assessed was red blood cells.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12
Vital signs assessed were systolic and diastolic blood pressure.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12
Vital signs assessed was heart rate.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12
Vital signs assessed was respiratory rate.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12
Vital signs assessed was temperature.
Measured: Baseline, Month 6 and Month 12
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12
Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12
ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.
Measured: Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12
ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.
Measured: Baseline, 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 posted30 Jun 2015 (estimated)
- Study start7 Jan 2016
- Primary completion14 Sep 2017
- Study completion14 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Capricor Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Awadalla, Study director, Capricor Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02485938
- Sponsor's protocol ID
- CAP-1002-DMD-01
- Acronym
- HOPE
- Status last verified
- Jan 2025
- First submitted
- 19 Jun 2015
- Last updated
- 9 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02485938
- Acronym / sponsor ID
- HOPE / CAP-1002-DMD-01
- Conditions
- Duchenne Muscular Dystrophy, Cardiomyopathy
- Keywords
- Duchenne Muscular Dystrophy, Cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Capricor Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Jan 2016 (actual)
- Primary completion
- 14 Sep 2017 (actual)
- Study completion
- 14 Sep 2017 (actual)
- First posted
- 30 Jun 2015 (estimated)
- Results
- Posted 9 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.
Detailed description
Approximately 24, and not more than 30, participants will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2.
The first 6-8 randomized participants will comprise Group 1, and will include a minimum of 3 participants completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused participants and for at least 6 participants overall.
Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 participants. Screening and randomization will continue until at total of 12 participants are infused with CAP 1002 or 30 participants are randomized into the study, whichever comes first.
All participants assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior).
Participants randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the participant on an ongoing basis, and will receive no infusion.
Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible participants randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible participants randomized to the usual care arm will occur 7 days after the date of randomization. All randomized participants will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Experimental | Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)CAP-1002 is an investigational product consisting of allogeneic cardiosphere-derived cells (CDCs). All subjects assigned to the active treatment arm will receive an intended total dose of 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). If any of the three coronary arteries are deemed by the infusing Investigator to supply less than 30% of the left ventricular myocardium, the infusing Investigator may choose to infuse only 12.5M cells into that coronary artery or arteries. Therefore the full dose of CAP-1002 delivered may range from 50M cells to 75M cells provided that all three arteries are infused. |
| Usual Care | No intervention | –Subjects randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the subject on an ongoing basis, and will receive no infusion. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Male participants 18 years of age or older must be able to provide informed consent and follow up with protocol procedures. Male participants at least 12 years of age but younger than 18 years of age must be able to provide assent with parent or guardian providing permission for study participation. Only male participants will be randomized into this study.
- Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.
- Cardiomyopathy with left ventricular scar by late gadolinium enhancement (LGE) in at least 4 segments as assessed by contrast-enhanced MRI and EF >35% at the time of screening.
- Use of evidence based medical-therapy in accordance with the "DMD Care Considerations Working Group" guidelines for the management of DMD, for at least three months prior to signing the consent form (or, providing assent) or documented contraindication or intolerance or patient preference.
- Participants must be taking systemic glucocorticoids for at least six months prior to screening.
Key exclusion criteria (27 listed)
- Therapy with intravenous inotropic or vasoactive medications at the time of screening.
- Inability to undergo cardiac catheterization and/or MRI without general anesthesia.
- Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling.
- Planned or likely major surgery in the next 12 months after planned randomization.
- Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD.
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 |
|---|---|---|
| Allogeneic Cardiosphere-Derived Cells (CAP-1002)Also: CAP-1002Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Drug | Allogeneic Cardiosphere-Derived Cells (CAP-1002) |
Primary outcome
Number of Participants Experiencing Any of the Adjudicated Events
Time frame: Within 72 hours post-infusion
Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations).
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Up to Month 12 post-infusion
An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.
Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Clinical chemistry parameters assessed were chloride, potassium and sodium.
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Clinical chemistry parameter assessed was albumin.
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Clinical chemistry parameter assessed was glucose.
Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Hematological parameter assessed was hemoglobin.
Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Hematological parameter assessed was red blood cells.
Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Vital signs assessed were systolic and diastolic blood pressure.
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Vital signs assessed was heart rate.
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Vital signs assessed was respiratory rate.
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Vital signs assessed was temperature.
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.
Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12
Time frame: Baseline, Month 6 and Month 12
ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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. This study is not recruiting at the moment.
Mark Awadalla study director
Capricor Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Cardiosphere-Derived Cells (CAP-1002) in people with Duchenne Muscular Dystrophy and Cardiomyopathy, run by Capricor Inc. 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 7 inclusion and 27 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: finished, as of the sponsor's last update on 9 Jan 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 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?
Yes. Results are posted in the registry (first posted 9 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Experiencing Any of the Adjudicated Events (time frame: Within 72 hours post-infusion). The study lists 15 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 9 Jan 2025.