Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy

NCT ID: NCT02256501

Last Updated: 2017-04-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-06-30
• Study Completion Date: 2016-12-31

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.

Conditions

Cardiomyopathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Mono Nunlear Cell (MNC)

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

Group Type: EXPERIMENTAL

Mono Nuclear Cell (MNC) transplantation

Intervention Type: BIOLOGICAL

Intracoronary administration of autologous bone marrow derived mononuclear cells

Control

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Mono Nuclear Cell (MNC) transplantation

Intracoronary administration of autologous bone marrow derived mononuclear cells

Intervention Type: BIOLOGICAL

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

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nasser Aghdami MD., PhD

OTHER_GOV

Sponsor Role: lead

Responsible Party

Nasser Aghdami MD., PhD

Hear of department of Regenerative Medicine of Royan Institute 7 cell therapy center

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Hamid Gourabi, PhD

Role: STUDY_CHAIR

Head of Royan Institute

Nasser Aghdami, MD,PhD

Role: STUDY_DIRECTOR

Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute

Mohammad Mahdavi, MD

Role: STUDY_DIRECTOR

Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran

Koorosh Vahidshahi, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran

Locations

Royan Institute

Tehran, , Iran

Countries

Iran

Related Links

http://Royaninstitute.org

Related Info

Other Identifiers

Royan-Heart-004

Identifier Type: -

Identifier Source: org_study_id