Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

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

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.

Study Overview

• Status: Completed
• Conditions: Cardiomyopathy
• Intervention / Treatment: Biological: Mono Nuclear Cell (MNC) transplantation

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Iran, Islamic Republic of
  • Tehran, Iran, Islamic Republic of
    • Royan Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mono Nunlear Cell (MNC); The patients with idiopathic dilated cardiomyopathy who underwent intracoronary injection of autologous bone marrow-derived mononuclear cells .	Biological: Mono Nuclear Cell (MNC) transplantation; Intracoronary administration of autologous bone marrow derived mononuclear cells
No Intervention: Control; The patients with cardiomyopathy that are under observe during the study.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Nasser Aghdami MD., PhD
• Investigators: Study Director: Mohammad Mahdavi, MD, Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran; Principal Investigator: Koorosh Vahidshahi, MD, Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2013
• Primary Completion (Actual): March 1, 2016
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: October 1, 2014
• First Submitted That Met QC Criteria: October 2, 2014
• First Posted (Estimate): October 3, 2014

Study Record Updates

• Last Update Posted (Actual): April 27, 2017
• Last Update Submitted That Met QC Criteria: April 26, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Keywords: Dilated cardiomyopathy pediatric heart failure cell therapy
• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Cardiomyopathies
• Other Study ID Numbers: Royan-Heart-004