Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS)

Phase 2 Study of Intracoronary Infusion of Cardiac Progenitor Cells in Patients With Univentricular Heart Disease

The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease. Patients with preoperative high-risk group or whose cardiac function did not recover postoperatively eventually have no choice other than heart transplantation.

Following the safety verification of the Phase I study (TICAP trial) that has been implemented and completed, the efficacy will be verified in this Phase II clinical study. The rationale of this study is based on the animal studies that transplanted cardiac progenitor cells may regenerate damaged myocardium by giving rise to cardiac muscle and vascular cell types. Preclinical and phase I studies indicate the possibilities of cardiac function improvements and reduced myocardial fibrosis by progenitor cell infusion.

The efficacy of cell transplantation is assessed as the primary endpoint of the Phase II clinical study. The improvements of cardiac ejection fraction assessed by echocardiography, ventriculography, and cardiac MRI, which are conducted before and after treatment, are assessed as the evaluation items. The absolute values of the improvement of cardiac ejection fraction obtained by the above three imaging modalities before and after treatment are compared between two groups of the transplantation group and non-transplantation group.

Inclusion Criteria:

1. Age: Age is 0 year or more and 20 years or less at the time of enrollment.
2. The patients have functional single ventricular physiology with the indication to have stage-2 or -3 palliative surgeries.
3. The ventricular ejection fraction <60%.

Exclusion Criteria:

1. Cardiogenic shock
2. A patient with unstoppable extracorporeal circulation
3. A patient with lethal, uncontrollable arrhythmia
4. A patient with a complication of coronary artery disease
5. A patient with a complication of brain dysfunction due to circulatory failure
6. A patient with malignant neoplasm
7. A patient with a complication of serious neurologic disorder
8. A patient with high-grade pulmonary embolism or pulmonary hypertension
9. A patient with high-grade renal failure
10. A patient with multiple organ failure
11. Active infection (including endocarditis)
12. Sepsis
13. Active hemorrhagic disease (e. g. gastrointestinal bleeding, injury)