Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy and safety of intracoronary injection of JRM-001 after reconstructive surgery in pediatric patients with functional single ventricle

Official Title
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Efficacy and Safety Study of Autologous Cardiac Stem Cells (JRM-001) Treated After Reconstructive Surgery in Pediatric Patients With Congenital Heart Disease: A Multicenter Randomized Single-blind Parallel-group Study

Conditions
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Hypoplastic Left Heart Syndrome, Single Ventricle

Intervention / Treatment
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* Genetic: Autologous cardiac stem cells (JRM-001)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Functional single ventricle patient with heart failure who is scheduled for stage 2 (Glenn) or stage 3 (Fontan) surgery EF(%) by echocardiography ≤ 55% Able to obtain written informed consent of participation in the study by a parent of the patient Exclusion Criteria: Known medical history of cardiogenic shock Lethal, uncontrollable arrhythmia Complication of coronary artery disease Eisenmenger syndrome Complication of brain dysfunction due to circulatory failure Malignant neoplasm Complication of severe neurologic disorder Severe pulmonary embolism or pulmonary hypertension Severe renal failure Multiple organ failure Active infection (including endocarditis) Sepsis Active hemorrhagic disease (e.g. gastrointestinal bleeding, injury) Known history of hypersensitivity to anti-infective drugs Inability to complete the protocol treatment and baseline to follow-up examinations

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 6 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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autologous cardiac stem cells (JRM-001)<br> | Genetic: Autologous cardiac stem cells (JRM-001)<br>* 3x 10^5 cells/kg, single treatment<br>|
| No Intervention: Usual care<br> | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in ejection fraction (EF(%)) assessed by MRI from baseline |  | Baseline, 6 and 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in EF(%) assessed by echocardiograms from baseline |  | Screening, Baseline, 3, 6 and 12 months | 
| Change in EF(%) assessed by cardiac catheterization from baseline |  | Baseline, 6 and 12 months | 
| Change in Ea/Ees assessed by cardiac catheterization from baseline |  | Baseline, 6 and 12 months | 
| Change in Ventricular Stiffness assessed by cardiac catheterization from baseline |  | Baseline, 6 and 12 months | 
| Change in heart failure index from baseline |  | Baseline, 3, 6 and 12 months | 
| Change in Quality of Life (QOL) index from baseline |  | Baseline, 6 and 12 months | 
| Number of adverse events |  | Up to 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cardiac stem/progenitor cells, autologous cell therapy, functional single ventricle, congenital heart disease