Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

Cardiomyopathy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02256501
• Other study ID #: Royan-Heart-004
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 1, 2014
• Last updated: April 26, 2017
• Start date: June 2013
• Est. completion date: December 2016

Study information

• Verified date: April 2017
• Source: Royan Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: December 2016
• Est. primary completion date: March 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 16 Years

Eligibility

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

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy
• Cardiomyopathy, Dilated

Intervention

Biological:
• Mono Nuclear Cell (MNC) transplantation
Intracoronary administration of autologous bone marrow derived mononuclear cells

Locations

Country	Name	City	State
Iran, Islamic Republic of	Royan Institute	Tehran

Sponsors (1)

Lead Sponsor	Collaborator
Nasser Aghdami MD., PhD

Country where clinical trial is conducted

Iran, Islamic Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Death	The rate of patients' death 3 months after cell transplantation	3months
Primary	Arrest	The rate of patients' arrests 3 months after cell transplantation	3 months
Secondary	Dysrhythmia	The rate of dysarthymia 3 months after cell transplantation	3 months
Secondary	Heart transplantation	The rate of demand for heart transplantation 3 months after cell transplantation.	3 months
Secondary	Hospital admission for heart failure	The rate of hospital administration 3 months after cell transplantation.	3 months

External Links

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