Dilated Cardiomyopathy
Conditions
Brief summary
A phase 1 prospective study to determine the procedural feasibility and safety and preliminary efficacy of intracoronary infusion of cardiosphere-derived cells (CDCs) in patients with dilated cardiomyopathy.
Detailed description
Five consecutive patients will be enrolled to a phase 1 study to verify the procedural feasibility and safety with the CDC infusion group.
Interventions
BIOLOGICALCDC infusion
Injection of CDCs (0.3 million per kg of body weight).
Sponsors
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Okayama University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Transcoronary infusion
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
* Patients diagnosed as dilated cardiomyopathy. * Patients aged under 18 years old. * Cardiac ejection fraction \< 40%.
Exclusion criteria
* Contradiction to cardiac magnetic resonance imaging. * Cardiogenic shock. * A patient with unstoppable extracorporeal circulation. * A patient with lethal, uncontrollable arrhythmia. * A patient with a complication of coronary artery disease. * A patient with a complication of brain dysfunction due to circulatory failure. * A patient with malignant neoplasm. * A patient with a complication of a serious neurologic disorder. * A patient with high-grade pulmonary embolism or pulmonary hypertension. * A patient with high-grade renal failure. * A patient with multiple organ failure. * Active infection (including endocarditis). * Sepsis. * Active hemorrhagic disease (e.g. gastrointestinal bleeding, injury).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs. | 6 months after CDC treatment | Assessment of major adverse cardiac events include death, sustained/symptomatic ventricular tachycardia, aggravation of heart failure, acute coronary syndrome, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Ejection Fraction | 6 months after protocol treatment | To determine the changes in cardiac function by ejection fraction with cardiac MRI evaluation in CDC-treated participants from baseline to 6 months of follow up. |
Countries
Japan
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| CDC Infusion CDCs infusion by coronary intervention.
CDC infusion: Injection of CDCs (0.3 million per kg of body weight). | 5 |
| Total | 5 |
Baseline characteristics
| Characteristic | CDC Infusion |
|---|---|
| Age, Continuous | 11.4 years STANDARD_DEVIATION 6.7 |
| Cardiac Function | 28.5 percentage of baseline ejection fraction STANDARD_DEVIATION 10.7 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 5 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 5 |
| other Total, other adverse events | 0 / 5 |
| serious Total, serious adverse events | 1 / 5 |
Outcome results
Primary
Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs.
Assessment of major adverse cardiac events include death, sustained/symptomatic ventricular tachycardia, aggravation of heart failure, acute coronary syndrome, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.
Time frame: 6 months after CDC treatment
Population: Adverse events
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CDC Infusion | Number of Participants With Major Cardiac Adverse Events Related to Transcoronary Infusion of CDCs. | 1 Participants |
Secondary
Change in Ejection Fraction
To determine the changes in cardiac function by ejection fraction with cardiac MRI evaluation in CDC-treated participants from baseline to 6 months of follow up.
Time frame: 6 months after protocol treatment
Population: Changes in cardiac function
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| CDC Infusion | Change in Ejection Fraction | 4.5 percentage of ejection fraction | Standard Deviation 10.2 |