Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

Study Overview
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Brief Summary
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According to the high morbidity and mortality of idiopathic Dilated CardioMyopathy (IDCM) in pediatric, new modality of treatment is emerging. There are some case reports of administration of stem cell therapy. The investigators design the first randomized clinical trial in this setting. The investigators enroll 32 pediatric IDCM patients in two groups (16 pts. in each group including cell therapy and control). The investigators assess the safety and efficacy of intracoronary transplantation of autologous bone marrow derived mononuclear cells in this patients compared to control group.

Detailed Description
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Dilated cardiomyopathy as the most common form of cardiomyopathy is a rare but life-threatening disorder in children. The primary cause of nearly 37 % of children with DCM was unknown at diagnosis. Despite of the developing in the medical and surgical treatment during the past several decades, standard treatments (including Digitalis, diuretics, inhibitors of ACE, beta blockers, antiplatelet drugs and treatments Antiarrhythmic), may stabilize the condition, but will not restore heart function to its previous condition. Therapy remains complex and expensive. For some not all children the heart transplantation is only option and mortality continues to be high, also. Stem cell and cell-based therapies offer an innovative approach to reverse cardiac structure and function towards normal, possibly reducing the need for aggressive therapies and cardiac transplantation. According to the inclusion and exclusion criteria of trial, 32 patients with left ventricular ejection fraction less than 45% who resistance to the standard medical therapy were randomly allocated in 2 groups including BM-derived mononuclear (n=16) and control (n=16). Only the MNC group underwent the bone marrow aspiration and intracoronary injection. The investigators followed all of patients at 2 weeks, 1, 2, 4 and 6 months after transplantation for cell therapy group or registration for placebo by physical examination, laboratory tests and imaging such as echocardiography, CXR and CMR.

Official Title
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Intracoronary Transplantation of Autologous Bone Marrow Derived Mononuclear Cells (MNC) in Idiopathic Dilated Cardiomyopathy in Pediatric Patients: Clinical Trial Phase I/II

Conditions
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Cardiomyopathy

Intervention / Treatment
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* Biological: Mono Nuclear Cell (MNC) transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age between 1-16 year(s) LVEF <45% (echocardiography) Duration of diagnosis more than 3 months Resistance to Standard therapy more than 2 months Informed consent Exclusion Criteria: Congenital Heart Disease Active infection less than one month Dysrhythmia Cardiogenic shock Renal failure Immune Deficiency (Documentation) Terminal illness or malignancy(Documentation) TORCH (Documentation) Metabolic disorder (Documentation) Neuromuscular disorder (Documentation) Autoimmune disease (Documentation) Developmental delay Cytotoxic drugs Previous bone marrow transplant Contraindications to CMR such as metallic implants

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 16 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: Mono Nunlear Cell (MNC)<br>The patients with idiopathic dilated cardiomyopathy who underwent intracoronary injection of autologous bone marrow-derived mononuclear cells . | Biological: Mono Nuclear Cell (MNC) transplantation<br>* Intracoronary administration of autologous bone marrow derived mononuclear cells<br>|
| No Intervention: Control<br>The patients with cardiomyopathy that are under observe during the study. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Death | The rate of patients' death 3 months after cell transplantation | 3months | 
| Arrest | The rate of patients' arrests 3 months after cell transplantation | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dysrhythmia | The rate of dysarthymia 3 months after cell transplantation | 3 months | 
| Heart transplantation | The rate of demand for heart transplantation 3 months after cell transplantation. | 3 months | 
| Hospital admission for heart failure | The rate of hospital administration 3 months after cell transplantation. | 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Dilated cardiomyopathy pediatric heart failure cell therapy