Primary Idiopathic Dilated CardiomyopathyPhase 1/2NCT01957826
Mesenchymal Stem Cells for Idiopathic Dilated Cardiomyopathy
A Phase 1/2 study testing bone marrow-derived MSCs injection in people with Primary Idiopathic Dilated Cardiomyopathy, run by Hospital General Universitario Gregorio Marañon at 2 sites in Spain.
Trial summary
The purpose of this study is to assess the safety, the feasibility and the efficacy of transendocardial injection of bone marrow-derived mesenchymal stem cells (MSCs) in patients with dilated idiopathic cardiomyopathy. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Primary Idiopathic Dilated Cardiomyopathy
- What is being tested?
- bone marrow-derived MSCs injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 2 sitesSpain
- Study dates
- Mar 2013to Mar 2018
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- II-III NYHA functional class, under optimal medical therapy.
- LVEF ≥ 20% and ≤ 45% by echocardiography, SPECT or left ventriculogram one month prior to enrollment.
- Anterior wall thickness ≥ 8 mm by echocardiography or MRI one month prior to enrollment.
- Idiopathic DCM diagnosis (having excluded CAD, valvular heart disease, cardiac toxic drugs, tachyarrhythmias, metabolic diseases, systemic diseases, infectious diseases) six months prior to enrollment.
- Patients rejected for heart transplantation should have been discussed in the Heart Team at their respective centres, and a document stating the reason for exclusion will be kept in the medical record.
- Able to exercise on a treadmill, MVO2 between ≥ 12 and ≤ 21 ml/Kg/min.
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- Hemodynamic stability (blood pressure > 100/40 mmHg, heart rate < 110 bpm and oxygen saturation > 95%).
- Negative pregnancy test in women.
- Signed informed consent
Who cannot join
- Evidence of secondary dilated cardiomyopathy causes: CAD, valvular heart disease, cardiac toxic drugs, tachyarrhythmias, metabolic diseases, systemic diseases, infectious diseases, myocarditis or postpartum ventricular dysfunction.
- Permanent atrial fibrillation.
- Candidates for ICD or CRT devices. Patients with theses devices can be enrolled if the device has been implanted at least 6 months before inclusion, and only if no-response has been observed to CRT.
- Candidates for heart transplantation if surgery is anticipated in the next 2 years.
- Left ventricular thrombus by echocardiography, MRI or left ventriculogram.
- Peripheral artery disease that precludes cardiac catheterization with 8 Fr sheaths. .
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- Anterior wall thickness < 8 mm by echocardiography or MRI one month prior to enrollment.
- Chronic renal failure (creatinine > 2,5 mg/dL).
- I or IV NYHA functional class. Cardiogenic shock is defined as systolic blood pressure < 90 mmHg with no response to fluids, or < 100 mmHg with inotropes and without bradycardia.
- Previous history of drug abuse (alcohol, etc…).
- Acute or chronic infectious disease (including B/C hepatitis and HIV).
- Pregnancy or child-bearing period.
- MRI contraindications: pacemakers, ICD, metalic prosthesis, etc.
- Bleeding or coagulation disorders (INR > 2 without anticoagulation treatment).
- Cancer history 5 years prior to enrollment.
- Life expectancy less than 1 year.
- Any disease or condition that the investigator finds decisive for exclusion
Trial locations and contacts
2 sites in 1 country.
Francisco Fernandez Avilés, MD contact
Spain2 sites, 2 recruiting
- Recruiting
Hospital General Universitario Gregorio Marañón
Madrid, Madrid, 28007
Ricardo Sanz, MD, 034 91 426 5882, rsanzruiz@hotmail.com
• Francisco Fernandez Aviles, PhD
- Not yet recruiting
Hospital Clinico Universitario de Valladolid
Valladolid, Valladolid
• José Alberto San Román, MD
Javier López
About this study
Official title: Phase I/II Randomized Clinical Trial to Assess the Safety and Feasibility of Transendocardial Injection of Bone Marrow Autologous Mesenchymal Stem Cells in Patients With Idiopathic Dilated Cardiomyopathy.
The purpose of this study is to assess the safety, the feasibility and the efficacy of transendocardial injection of bone marrow-derived mesenchymal stem cells (MSCs) in patients with dilated idiopathic cardiomyopathy.
Read the detailed description
Chronic congestive heart failure (CHF) is a public health problem that entails high rates of morbidity and mortality, and enormous costs for health systems worldwide. In the United States there are 5 million people living with CHF, and each year 60.000 people reach terminal phases of the disease, with mortality rates of 70-80% at two years. Although the first cause of CHF in developed countries is atherosclerotic coronary artery disease (CAD), dilated idiopathic cardiomyopathy (DCM) represents almost half of the cases of newly diagnosed CHF. Treatment of CHF includes pharmacological and non-pharmacological strategies, including implantable cardioverter defibrillators, cardiac resynchronization therapy and heart transplantation. Despite all these advances, CHF prognosis remains poor. Cardiac stem cell therapy emerged more than ten years ago as a new hope for CHF patients.
Although the most extensive evidence of the benefits of stem cell therapy for cardiovascular diseases refers to ischemic heart disease (CAD), initial experiences with stem cells for other conditions such as DCM are encouraging.
This randomized clinical trial will include 70 patients with DCM, left ventricular ejection fraction (LVEF) between 20% and 45%, and who are symptomatic in New York Heart Association (NYHA) functional class II-III/IV. In a first-in-man pilot phase, 10 patients will be treated with transendocardial injections of bone marrow-derived MSCs after cardiac catheterization and NOGA XPTM mapping of the left ventricle. A Data and Safety Monitoring Board (DSMB) will analyse the safety and feasibility of this first phase of the trial, and then 60 patients more will be randomized to receive MSCs or placebo (ratio 3:1).
Primary objectives include safety and feasibility variables, and secondary objectives include efficacy variables. All patients will be studied with a complete cardiac imaging protocol that includes: electrocardiography, echocardiography, treadmill tests with oxygen consumption, holter, laboratory analyses, magnetic resonance imaging (MRI), single photon emission computed tomography (SPECT), electromechanical mapping (NOGA XPTM) and quality of life questionnaires.
- Being tested
- bone marrow-derived MSCs injection, placebo intervention
- Keywords
- mesenchymal, stem cells, transendocardial injection, dilated idiopathic cardiomyopathy, idiopathic cardiomyopathy
- Medical subject headings
- Cardiomyopathy, Dilated, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
bone marrow-derived MSCs injectionOther
transendocardial injection of 30-40 million bone marrow-derived MSCs with the NOGA XPTM platform. 15 injections in the anterior wall of the left ventricle.
placebo interventionOther
placebo administration
| Study group | Role | What they receive |
|---|---|---|
| placebo comparator | Placebo comparator | transendocardial injection of placebo solution Receives: Other: placebo intervention |
| bone marrow-derived MSCs injection | Experimental | transendocardial injection of 30-40 million bone marrow-derived MSCs with the NOGA XPTM platform. 15 injections in the anterior wall of the left ventricle. Receives: Other: bone marrow-derived MSCs injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Major adverse cardiac adverse events. SAEs and AEs.
Major adverse cardiac adverse events includes cerebral adverse events
Measured: change from enrollment( 1, 3, 6, 12, 18 and 24 months)
NYHA functional class.
Measured: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
Incidence of complications with the use of NOGA XPTM catheters.
Measured: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
Laboratory parameters including C-reactive protein an brain natriuretic peptide
Measured: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
Secondary outcomes (6)
NYHA Functional Class
Measured: 1, 3, 6, 12, 18, 24 months
Max.oxygen consumption(MVO2),functional capacity.
Measured: 6,12,24 months
Quality of life questionnaires
include 36-item Short Form Survey(SF 36) and Minnesota Living UIT Heart Failure questionnaire
Measured: 6,12 and 24 months
Extension. of perfusion defects(MRI/SPECT).
Measured: 6 and 24 months
LVEF, ventricular vol.,wall motion score index(echocard./MRI/SPECT
Measured: 6,12 and 24 months
LVEF(left ventriculogram, electromech. mapping parameters(NOGA XPTM))
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2013 (estimated)
- Study startMar 2013
- Primary completionMar 2018 (estimated)
- Study completionMar 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Idiopathic Dilated 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital General Universitario Gregorio Marañon
- Sponsor type
- University, hospital or other
- Collaborators
- Ministerio de Sanidad, Servicios Sociales e Igualdad
- Responsible party
- Sponsor
- Investigators
- Francisco Fernandez Aviles, PhD, Study chair, Hospital General Universitario Gregorio Marañon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01957826
- Sponsor's protocol ID
- FIBHGM-ECNC017-2010
- Other IDs
- 2010-024406-35, MIYOCYTE
- Status last verified
- Nov 2016
- First submitted
- 21 Dec 2012
- Last updated
- 18 Nov 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Randomized Clinical Trial to Assess the Safety and Feasibility of Transendocardial Injection of Bone Marrow Autologous Mesenchymal Stem Cells in Patients With Idiopathic Dilated Cardiomyopathy.
- ClinicalTrials.gov ID
- NCT01957826
- Acronym / sponsor ID
- FIBHGM-ECNC017-2010
- Keywords
- mesenchymal, stem cells, transendocardial injection, dilated idiopathic cardiomyopathy, idiopathic cardiomyopathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Hospital General Universitario Gregorio MarañonUniversity, hospital or other
- Collaborators
- Ministerio de Sanidad, Servicios Sociales e Igualdad
- Study start
- Mar 2013
- Primary completion
- Mar 2018 (estimated)
- Study completion
- Mar 2018 (estimated)
- First posted
- 8 Oct 2013 (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.
The purpose of this study is to assess the safety, the feasibility and the efficacy of transendocardial injection of bone marrow-derived mesenchymal stem cells (MSCs) in patients with dilated idiopathic cardiomyopathy.
Detailed description
Chronic congestive heart failure (CHF) is a public health problem that entails high rates of morbidity and mortality, and enormous costs for health systems worldwide. In the United States there are 5 million people living with CHF, and each year 60.000 people reach terminal phases of the disease, with mortality rates of 70-80% at two years. Although the first cause of CHF in developed countries is atherosclerotic coronary artery disease (CAD), dilated idiopathic cardiomyopathy (DCM) represents almost half of the cases of newly diagnosed CHF. Treatment of CHF includes pharmacological and non-pharmacological strategies, including implantable cardioverter defibrillators, cardiac resynchronization therapy and heart transplantation. Despite all these advances, CHF prognosis remains poor. Cardiac stem cell therapy emerged more than ten years ago as a new hope for CHF patients.
Although the most extensive evidence of the benefits of stem cell therapy for cardiovascular diseases refers to ischemic heart disease (CAD), initial experiences with stem cells for other conditions such as DCM are encouraging.
This randomized clinical trial will include 70 patients with DCM, left ventricular ejection fraction (LVEF) between 20% and 45%, and who are symptomatic in New York Heart Association (NYHA) functional class II-III/IV. In a first-in-man pilot phase, 10 patients will be treated with transendocardial injections of bone marrow-derived MSCs after cardiac catheterization and NOGA XPTM mapping of the left ventricle. A Data and Safety Monitoring Board (DSMB) will analyse the safety and feasibility of this first phase of the trial, and then 60 patients more will be randomized to receive MSCs or placebo (ratio 3:1).
Primary objectives include safety and feasibility variables, and secondary objectives include efficacy variables. All patients will be studied with a complete cardiac imaging protocol that includes: electrocardiography, echocardiography, treadmill tests with oxygen consumption, holter, laboratory analyses, magnetic resonance imaging (MRI), single photon emission computed tomography (SPECT), electromechanical mapping (NOGA XPTM) and quality of life questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| placebo comparator | Placebo comparator | Other: placebo interventiontransendocardial injection of placebo solution |
| bone marrow-derived MSCs injection | Experimental | Other: bone marrow-derived MSCs injectiontransendocardial injection of 30-40 million bone marrow-derived MSCs with the NOGA XPTM platform. 15 injections in the anterior wall of the left ventricle. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- II-III NYHA functional class, under optimal medical therapy.
- LVEF ≥ 20% and ≤ 45% by echocardiography, SPECT or left ventriculogram one month prior to enrollment.
- Anterior wall thickness ≥ 8 mm by echocardiography or MRI one month prior to enrollment.
- Idiopathic DCM diagnosis (having excluded CAD, valvular heart disease, cardiac toxic drugs, tachyarrhythmias, metabolic diseases, systemic diseases, infectious diseases) six months prior to enrollment.
- Patients rejected for heart transplantation should have been discussed in the Heart Team at their respective centres, and a document stating the reason for exclusion will be kept in the medical record.
Key exclusion criteria (17 listed)
- Evidence of secondary dilated cardiomyopathy causes: CAD, valvular heart disease, cardiac toxic drugs, tachyarrhythmias, metabolic diseases, systemic diseases, infectious diseases, myocarditis or postpartum ventricular dysfunction.
- Permanent atrial fibrillation.
- Candidates for ICD or CRT devices. Patients with theses devices can be enrolled if the device has been implanted at least 6 months before inclusion, and only if no-response has been observed to CRT.
- Candidates for heart transplantation if surgery is anticipated in the next 2 years.
- Left ventricular thrombus by echocardiography, MRI or left ventriculogram.
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 |
|---|---|---|
| bone marrow-derived MSCs injectiontransendocardial injection of 30-40 million bone marrow-derived MSCs with the NOGA XPTM platform. 15 injections in the anterior wall of the left ventricle. | Other | bone marrow-derived MSCs injection |
| placebo interventionplacebo administration | Other | placebo comparator |
Primary outcome
Major adverse cardiac adverse events. SAEs and AEs.
Time frame: change from enrollment( 1, 3, 6, 12, 18 and 24 months)
Major adverse cardiac adverse events includes cerebral adverse events
NYHA functional class.
Time frame: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
Incidence of complications with the use of NOGA XPTM catheters.
Time frame: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
Laboratory parameters including C-reactive protein an brain natriuretic peptide
Time frame: Change from enrolment( 1, 3, 6, 12, 18, 24 months)
6 secondary outcomes
NYHA Functional Class
Time frame: 1, 3, 6, 12, 18, 24 months
Max.oxygen consumption(MVO2),functional capacity.
Time frame: 6,12,24 months
Quality of life questionnaires
Time frame: 6,12 and 24 months
Extension. of perfusion defects(MRI/SPECT).
Time frame: 6 and 24 months
LVEF, ventricular vol.,wall motion score index(echocard./MRI/SPECT
Time frame: 6,12 and 24 months
LVEF(left ventriculogram, electromech. mapping parameters(NOGA XPTM))
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2016.
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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.
Francisco Fernandez Avilés, MD contact
Francisco Fernandez Aviles, PhD study chair
Hospital General Universitario Gregorio Marañon
2 sites 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 bone marrow-derived MSCs injection in people with Primary Idiopathic Dilated Cardiomyopathy, run by Hospital General Universitario Gregorio Marañon at 2 sites in Spain.
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 9 inclusion and 17 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: status not confirmed, as of the sponsor's last update on 18 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Spain. 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. Some participants in this study may receive a placebo. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ricardo Sanz, MD (034 91 426 5882, rsanzruiz@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Major adverse cardiac adverse events. SAEs and AEs. (time frame: change from enrollment( 1, 3, 6, 12, 18 and 24 months)). The study lists 4 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 18 Nov 2016.