CardiomyopathyPhase 1/2NCT02256501
Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy
A Phase 1/2 study testing Mono Nuclear Cell (MNC) transplantation in people with Cardiomyopathy, run by Nasser Aghdami MD., PhD at 1 site in Iran.
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. Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Cardiomyopathy
- What is being tested?
- Mono Nuclear Cell (MNC) transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteIran
- Study dates
- Jun 2013to Dec 2016
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age between 1-16 year(s)
- LVEF <45% (echocardiography)
- Duration of diagnosis more than 3 months
- Resistance to Standard therapy more than 2 months
- Informed consent
Who cannot join
- Congenital Heart Disease
- Active infection less than one month
- Dysrhythmia
- Cardiogenic shock
- Renal failure
- Immune Deficiency (Documentation)
Show all 15Show fewer
- Terminal illness or malignancy(Documentation)
- TORCH (Documentation)
- Metabolic disorder (Documentation)
- Neuromuscular disorder (Documentation)
- Autoimmune disease (Documentation)
- Developmental delay
- Cytotoxic drugs
- Previous bone marrow transplant
- Contraindications to CMR such as metallic implants
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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
- Being tested
- Mono Nuclear Cell (MNC) transplantation
- Keywords
- Dilated cardiomyopathy pediatric heart failure cell therapy
- Medical subject headings
- Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mono Nuclear Cell (MNC) transplantationBiological
Intracoronary administration of autologous bone marrow derived mononuclear cells
| Study group | Role | What they receive |
|---|---|---|
| Mono Nunlear Cell (MNC) | Experimental | The patients with idiopathic dilated cardiomyopathy who underwent intracoronary injection of autologous bone marrow-derived mononuclear cells . Receives: Biological: Mono Nuclear Cell (MNC) transplantation |
| Control | No intervention | The patients with cardiomyopathy that are under observe during the study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Death
The rate of patients' death 3 months after cell transplantation
Measured: 3months
Arrest
The rate of patients' arrests 3 months after cell transplantation
Measured: 3 months
Secondary outcomes (3)
Dysrhythmia
The rate of dysarthymia 3 months after cell transplantation
Measured: 3 months
Heart transplantation
The rate of demand for heart transplantation 3 months after cell transplantation.
Measured: 3 months
Hospital admission for heart failure
The rate of hospital administration 3 months after cell transplantation.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2014 (estimated)
- Study startJun 2013
- Primary completionMar 2016
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nasser Aghdami MD., PhD
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Nasser Aghdami MD., PhD, Royan Institute
- Investigators
- Hamid Gourabi, PhD, Study chair, Head of Royan InstituteNasser Aghdami, MD,PhD, Study director, Head of Department of Regenerative Medicine & Cell therapy center of Royan InstituteMohammad Mahdavi, MD, Study director, Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, IranKoorosh Vahidshahi, MD, Principal investigator, Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02256501
- Sponsor's protocol ID
- Royan-Heart-004
- Status last verified
- Apr 2017
- First submitted
- 1 Oct 2014
- Last updated
- 27 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intracoronary Transplantation of Autologous Bone Marrow Derived Mononuclear Cells (MNC) in Idiopathic Dilated Cardiomyopathy in Pediatric Patients: Clinical Trial Phase I/II
- ClinicalTrials.gov ID
- NCT02256501
- Acronym / sponsor ID
- Royan-Heart-004
- Conditions
- Cardiomyopathy
- Keywords
- Dilated cardiomyopathy pediatric heart failure cell therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Nasser Aghdami MD., PhDOther government
- Study start
- Jun 2013 (actual)
- Primary completion
- Mar 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 3 Oct 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mono Nunlear Cell (MNC) | Experimental | Biological: Mono Nuclear Cell (MNC) transplantationThe patients with idiopathic dilated cardiomyopathy who underwent intracoronary injection of autologous bone marrow-derived mononuclear cells . |
| Control | No intervention | –The patients with cardiomyopathy that are under observe during the study. |
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Age between 1-16 year(s)
- LVEF <45% (echocardiography)
- Duration of diagnosis more than 3 months
- Resistance to Standard therapy more than 2 months
- Informed consent
Key exclusion criteria (15 listed)
- Congenital Heart Disease
- Active infection less than one month
- Dysrhythmia
- Cardiogenic shock
- Renal failure
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mono Nuclear Cell (MNC) transplantationIntracoronary administration of autologous bone marrow derived mononuclear cells | Biological | Mono Nunlear Cell (MNC) |
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
3 secondary outcomes
Dysrhythmia
Time frame: 3 months
Heart transplantation
Time frame: 3 months
Hospital admission for heart failure
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2017.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hamid Gourabi, PhD study chair
Head of Royan Institute
Nasser Aghdami, MD,PhD study director
Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
Mohammad Mahdavi, MD study director
Department of Pediatric Cardiology, Rajaie Cardiovascular Medical and Research Center, Iran University of Medical Sciences, Tehran, Iran
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Mono Nuclear Cell (MNC) transplantation in people with Cardiomyopathy, run by Nasser Aghdami MD., PhD at 1 site in Iran.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Iran. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Death (time frame: 3months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2017.