Clinical Trial: Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary: 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.
Sponsor: Nasser Aghdami MD., PhD

Current Primary Outcome:

• Death [ Time Frame: 3months ]
  The rate of patients' death 3 months after cell transplantation
• Arrest [ Time Frame: 3 months ]
  The rate of patients' arrests 3 months after cell transplantation

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Dysrhythmia [ Time Frame: 3 months ]
  The rate of dysarthymia 3 months after cell transplantation
• Heart transplantation [ Time Frame: 3 months ]
  The rate of demand for heart transplantation 3 months after cell transplantation.
• Hospital admission for heart failure [ Time Frame: 3 months ]
  The rate of hospital administration 3 months after cell transplantation.

Original Secondary Outcome: Same as current

Information By: Royan Institute

Dates:
Date Received: October 1, 2014
Date Started: June 2013
Date Completion:
Last Updated: April 26, 2017
Last Verified: April 2017