Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy

Study Overview
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Brief Summary
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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
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Five consecutive patients will be enrolled to a phase 1 study to verify the procedural feasibility and safety with the CDC infusion group.

Official Title
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A Prospective Phase 1 Trial of Cardiac Progenitor Cell Therapy in Children With Dilated Cardiomyopathy

Conditions
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Dilated Cardiomyopathy

Intervention / Treatment
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* Biological: CDC infusion


Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Transcoronary infusion
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CDC infusion<br>CDCs infusion by coronary intervention. | Biological: CDC infusion<br>* Injection of CDCs (0.3 million per kg of body weight).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 6 months after CDC treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 6 months after protocol treatment |