Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

Nasser Aghdami MD., PhD

Status and phase: Completed; Phase 2; Phase 1

Conditions: Cardiomyopathy

Treatments: Biological: Mono Nuclear Cell (MNC) transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT02256501

Royan-Heart-004

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 30 patients

Sex: All

Ages: 1 to 16 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age between 1-16 year(s)
2. LVEF <45% (echocardiography)
3. Duration of diagnosis more than 3 months
4. Resistance to Standard therapy more than 2 months
5. Informed consent

Exclusion criteria

1. Congenital Heart Disease
2. Active infection less than one month
3. Dysrhythmia
4. Cardiogenic shock
5. Renal failure
6. Immune Deficiency (Documentation)
7. Terminal illness or malignancy(Documentation)
8. TORCH (Documentation)
9. Metabolic disorder (Documentation)
10. Neuromuscular disorder (Documentation)
11. Autoimmune disease (Documentation)
12. Developmental delay
13. Cytotoxic drugs
14. Previous bone marrow transplant
15. Contraindications to CMR such as metallic implants

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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30 participants in 2 patient groups

Mono Nunlear Cell (MNC)

Experimental group

Description:

The patients with idiopathic dilated cardiomyopathy who underwent intracoronary injection of autologous bone marrow-derived mononuclear cells .

Treatment:

Biological: Mono Nuclear Cell (MNC) transplantation

Control

No Intervention group

Description:

The patients with cardiomyopathy that are under observe during the study.