CardiomyopathyPhase 1NCT02408432
Bone Marrow Derived Mesenchymal Stem Cells in Improving Heart Function in Patients With Heart Failure Caused by Anthracyclines
A Phase 1 study testing Laboratory Biomarker Analysis and Mesenchymal Stem Cell Transplantation in people with Cardiomyopathy and Heart Failure, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This randomized pilot phase I trial studies the side effects and best method of delivery of bone marrow derived mesenchymal stem cells (MSCs) in improving heart function in patients with heart failure caused by anthracyclines (a type of chemotherapy drug used in cancer treatment). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cardiomyopathy, Heart Failure
- What is being tested?
- Laboratory Biomarker Analysis and Mesenchymal Stem Cell Transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jan 2016to 8 Dec 2021
- Phase
- Phase 1
- 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
- Patients with LVEF =< 40% from treatment with anthracyclines for all malignancies at any dose at any time without evidence of other causes of cardiomyopathy
- Documented New York Heart Association (NYHA) class I, II and III
- Been treated with appropriate maximal medical therapy for heart failure
- Able to perform 6 minute walk test
- Patient or legally authorized representative able to sign informed consent
- Patients with persistent LV dysfunction 90 days after discontinuation of trastuzumab
Who cannot join
- Evidence of ischemic heart disease as determined by study cardiologist
- Significant valvular disease; (aortic stenosis [AS] with aortic valve area [AVA] < 1.5 and severe aortic regurgitation [AR] and mitral regurgitation [MR])
- History of familial cardiomyopathy
- Recent documented myocarditis within 2 months of consent
- History of infiltrative cardiomyopathy or restrictive cardiomyopathy
- Epidermal growth factor receptor (eGFR) < 50 by Mayo or Cockcroft formula
Show all 17Show fewer
- Liver function tests > 3 x upper limit of normal
- NYHA class IV heart failure
- Inotropic dependence
- Unstable or life-threatening arrhythmia
- Coagulopathy international normalized ratio (INR) > 1.5
- Mechanical or bioprosthetic heart valve
- Cardiogenic shock
- Breast feeding and/or pregnant women
- Autoimmune disorders on current immunosuppressive therapy
- Active infection not responding to appropriate therapy as determined by study chair
- Trastuzumab treatment within the last 3 months
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Intravenous Administration of Allogeneic Bone Marrow Derived Multipotent Mesenchymal Stromal Cells (MSCs) in Patients With Recent Onset Anthracycline-Associated Cardiomyopathy
This randomized pilot phase I trial studies the side effects and best method of delivery of bone marrow derived mesenchymal stem cells (MSCs) in improving heart function in patients with heart failure caused by anthracyclines (a type of chemotherapy drug used in cancer treatment). MSCs are a type of stem cell that can be removed from bone marrow and grown into many different cell types that can be used to treat cancer and other diseases, such as heart failure. Bone marrow derived MSCs may promote heart muscle cells repair and lead to reverse remodeling and ultimately improve heart function and decrease morbidity and mortality from progression to advanced heart failure.
Read the detailed description
PRIMARY OBJECTIVE:
- To demonstrate the safety of allogeneic human mesenchymal stem cells (hMSCs) administered by intravenous infusion in patients with left ventricular (LV) dysfunction and heart failure secondary to chemotherapy with anthracyclines.
SECONDARY OBJECTIVE:
- To demonstrate the efficacy of allogeneic hMSCs administered by intravenous infusion in patients with left ventricular dysfunction (left ventricular ejection fraction [LVEF] < 40%) and heart failure secondary to treatment with anthracyclines.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM I: Patients receive allogeneic hMSCs intravenously (IV) over 10-20 minutes once weekly for 4 weeks and standard of care drugs for heart failure.
ARM II: Patients receive only standard of care drugs for heart failure.
After completion of study treatment, patients are followed up monthly for 6 months and then at 12 months.
- Conditions
- Cardiomyopathy, Heart Failure
- Being tested
- Laboratory Biomarker Analysis, Standard of Care, Mesenchymal Stem Cell Transplantation
- Medical subject headings
- Cardiomyopathies, Heart Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory Biomarker AnalysisOther
Correlative studies
Standard of CareOther
Undergo mesenchymal stem cell infusion
Mesenchymal Stem Cell TransplantationDrug
Undergo mesenchymal stem cell infusion
| Study group | Role | What they receive |
|---|---|---|
| Arm I (hMSCs) | Experimental | Patients receive allogeneic hMSCs IV over 10-20 minutes once weekly for 4 weeks and standard of care drugs for heart failure. Receives: Other: Laboratory Biomarker Analysis; Other: Standard of Care; Drug: Mesenchymal Stem Cell Transplantation |
| Arm II (standard of care drugs) | Active comparator | Patients receive only standard of care drugs for heart failure. Receives: Other: Laboratory Biomarker Analysis; Other: Standard of Care; Drug: Mesenchymal Stem Cell Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events of intravenous injection of human mesenchymal stem cells (hMSCs) in patients with recent onset left ventricular systolic dysfunction from anthracyclines
Statistical analyses of safety will be descriptive. In particular, will provide a table of adverse events.
Measured: Up to 6 months
Secondary outcomes (10)
Change in improvement in left ventricular systolic function by 7 points
Change from baseline left ventricular ejection fraction (LVEF) at 6 months after randomization, expressed as a percentage. The comparison will be between the two groups of patients.
Measured: Baseline up to 6 months
Development of acute heart failure decompensation
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Emergency center visits for heart failure
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Need for new pacemaker/automatic implantable cardioverter defibrillator
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
New onset arrhythmias
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Sudden cardiac death
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Acute pulmonary edema
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Asymptomatic decrease in left ventricular ejection fraction (LVEF) of > 10%
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Baseline to up to 6 months
Heart failure admission
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Progression free survival
As regards statistical analyses, the results of the trial will be displayed in table format. Will provide confidence intervals of the differences in change from baseline between investigational and control groups. These intervals and the associate p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2015 (estimated)
- Study start11 Jan 2016
- Primary completion8 Dec 2021
- Study completion8 Dec 2021
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.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Amanda Olson, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02408432
- Sponsor's protocol ID
- 2014-0519
- Other IDs
- NCI-2015-00969, 2014-0519
- Status last verified
- May 2022
- First submitted
- 31 Mar 2015
- Last updated
- 17 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous Administration of Allogeneic Bone Marrow Derived Multipotent Mesenchymal Stromal Cells (MSCs) in Patients With Recent Onset Anthracycline-Associated Cardiomyopathy
- ClinicalTrials.gov ID
- NCT02408432
- Acronym / sponsor ID
- 2014-0519
- Conditions
- Cardiomyopathy, Heart Failure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 11 Jan 2016 (actual)
- Primary completion
- 8 Dec 2021 (actual)
- Study completion
- 8 Dec 2021 (actual)
- First posted
- 3 Apr 2015 (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.
This randomized pilot phase I trial studies the side effects and best method of delivery of bone marrow derived mesenchymal stem cells (MSCs) in improving heart function in patients with heart failure caused by anthracyclines (a type of chemotherapy drug used in cancer treatment). MSCs are a type of stem cell that can be removed from bone marrow and grown into many different cell types that can be used to treat cancer and other diseases, such as heart failure. Bone marrow derived MSCs may promote heart muscle cells repair and lead to reverse remodeling and ultimately improve heart function and decrease morbidity and mortality from progression to advanced heart failure.
Detailed description
PRIMARY OBJECTIVE:
- To demonstrate the safety of allogeneic human mesenchymal stem cells (hMSCs) administered by intravenous infusion in patients with left ventricular (LV) dysfunction and heart failure secondary to chemotherapy with anthracyclines.
SECONDARY OBJECTIVE:
- To demonstrate the efficacy of allogeneic hMSCs administered by intravenous infusion in patients with left ventricular dysfunction (left ventricular ejection fraction [LVEF] < 40%) and heart failure secondary to treatment with anthracyclines.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM I: Patients receive allogeneic hMSCs intravenously (IV) over 10-20 minutes once weekly for 4 weeks and standard of care drugs for heart failure.
ARM II: Patients receive only standard of care drugs for heart failure.
After completion of study treatment, patients are followed up monthly for 6 months and then at 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (hMSCs) | Experimental | Other: Laboratory Biomarker Analysis, Other: Standard of Care, Drug: Mesenchymal Stem Cell TransplantationPatients receive allogeneic hMSCs IV over 10-20 minutes once weekly for 4 weeks and standard of care drugs for heart failure. |
| Arm II (standard of care drugs) | Active comparator | Other: Laboratory Biomarker Analysis, Other: Standard of Care, Drug: Mesenchymal Stem Cell TransplantationPatients receive only standard of care drugs for heart failure. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients with LVEF =< 40% from treatment with anthracyclines for all malignancies at any dose at any time without evidence of other causes of cardiomyopathy
- Documented New York Heart Association (NYHA) class I, II and III
- Been treated with appropriate maximal medical therapy for heart failure
- Able to perform 6 minute walk test
- Patient or legally authorized representative able to sign informed consent
Key exclusion criteria (17 listed)
- Evidence of ischemic heart disease as determined by study cardiologist
- Significant valvular disease; (aortic stenosis [AS] with aortic valve area [AVA] < 1.5 and severe aortic regurgitation [AR] and mitral regurgitation [MR])
- History of familial cardiomyopathy
- Recent documented myocarditis within 2 months of consent
- History of infiltrative cardiomyopathy or restrictive cardiomyopathy
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 |
|---|---|---|
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (hMSCs), Arm II (standard of care drugs) |
| Standard of CareUndergo mesenchymal stem cell infusion | Other | Arm I (hMSCs), Arm II (standard of care drugs) |
| Mesenchymal Stem Cell TransplantationUndergo mesenchymal stem cell infusion | Drug | Arm I (hMSCs), Arm II (standard of care drugs) |
Primary outcome
Incidence of adverse events of intravenous injection of human mesenchymal stem cells (hMSCs) in patients with recent onset left ventricular systolic dysfunction from anthracyclines
Time frame: Up to 6 months
Statistical analyses of safety will be descriptive. In particular, will provide a table of adverse events.
10 secondary outcomes
Change in improvement in left ventricular systolic function by 7 points
Time frame: Baseline up to 6 months
Development of acute heart failure decompensation
Time frame: Up to 6 months
Emergency center visits for heart failure
Time frame: Up to 6 months
Need for new pacemaker/automatic implantable cardioverter defibrillator
Time frame: Up to 6 months
New onset arrhythmias
Time frame: Up to 6 months
Sudden cardiac death
Time frame: Up to 6 months
Acute pulmonary edema
Time frame: Up to 6 months
Asymptomatic decrease in left ventricular ejection fraction (LVEF) of > 10%
Time frame: Baseline to up to 6 months
Heart failure admission
Time frame: Up to 6 months
Progression free survival
Time frame: Up to 12 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 17 May 2022.
- MD Anderson Cancer Center WebsiteLink 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.
Amanda Olson principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Laboratory Biomarker Analysis and Mesenchymal Stem Cell Transplantation in people with Cardiomyopathy and Heart Failure, run by M.D. Anderson Cancer Center at 1 site in United States.
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 6 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: finished, as of the sponsor's last update on 17 May 2022. 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 United States. 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 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.
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?
Incidence of adverse events of intravenous injection of human mesenchymal stem cells (hMSCs) in patients with recent onset left ventricular systolic dysfunction from anthracyclines (time frame: Up to 6 months). 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 17 May 2022.