Clinical trial NCT02256501: Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy
Official title: Intracoronary Transplantation of Autologous Bone Marrow Derived Mononuclear Cells (MNC) in Idiopathic Dilated Cardiomyopathy in Pediatric Patients: Clinical Trial Phase I/II
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Cardiomyopathy
Keywords: Dilated cardiomyopathy pediatric heart failure cell therapy
Lead sponsor: Royan Institute
Sponsor class: OTHER_GOV
First posted: 2014-10-03
Enrollment: 30
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Death
timeFrame: 3months
measure: Arrest
timeFrame: 3 months
Secondary outcomes:
measure: Dysrhythmia
timeFrame: 3 months
measure: Heart transplantation
timeFrame: 3 months
measure: Hospital admission for heart failure
timeFrame: 3 months
Other outcomes:
Locations:
city: Tehran
country: Iran, Islamic Republic of
facility: Royan Institute
Study officials:
affiliation: Head of Royan Institute
name: Hamid Gourabi, PhD
role: STUDY_CHAIR
affiliation: Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
name: Nasser Aghdami, MD,PhD
role: STUDY_DIRECTOR
affiliation: Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran
name: Mohammad Mahdavi, MD
role: STUDY_DIRECTOR
affiliation: Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran
name: Koorosh Vahidshahi, MD
role: PRINCIPAL_INVESTIGATOR
References: