CardiomyopathyPhase 1NCT02962661
Donor Bone Marrow Derived Mesenchymal Stem Cells in Controlling Heart Failure in Patients With Cardiomyopathy Caused by Anthracyclines
A Phase 1 study testing Best Practice, 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 of donor bone marrow derived mesenchymal stem cells in controlling heart failure in patients with cardiomyopathy caused by anthracyclines. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cardiomyopathy, Heart Failure, Hematopoietic and Lymphoid Cell Neoplasm
- What is being tested?
- Best Practice, 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
- 72 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jul 2020to 30 Jul 2026
- 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% documented from treatment with anthracyclines for any malignancy at any dose at any time without evidence of other causes of cardiomyopathy.
- Age >/= 18 and </= 90 years of age. English and non-English speaking patients are eligible.
- Documented NYHA class I, II and III.
- For patients who have received trastuzumab: Persistent LV dysfunction must be present 90 days after discontinuation of trastuzumab.
- Able to perform 6 minute walk test.
- Been treated with appropriate maximal medical therapy for heart failure.
- Patient or legally authorized representative able to sign informed consent.
Who cannot join
- Evidence of Ischemic Heart Disease as determined by study cardiologist.
- Significant Valvular Disease. (AS with AVA <1.5 and severe AR and MR)
- History of Familial Cardiomyopathy.
- Recent documented myocarditis within 2 months of enrollment.
- History of Infiltrative cardiomyopathy or restrictive cardiomyopathy.
- Presence of left ventricular thrombus as documented by echocardiography or left ventriculogram.
Show all 19Show fewer
- Liver function tests > 3 x upper limit of normal.
- NYHA class IV heart failure.
- Inotropic dependence.
- Unstable or life-threatening arrhythmia.
- For patients not on anticoagulants, INR>1.5
- Mechanical or Bioprosthetic heart valve.
- Cardiogenic shock.
- Breastfeeding 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.
- Automatic implantable cardioverter defibrillator (AICD) placement within the last 30 days.
- AICD firing within the last 30 days.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomized 3-Arm Trial With Standard of Care Alone vs Either Intravenous Infusion or Transendocardial Injection of Allogeneic Bone Marrow Derived Multipotent Mesenchymal Stromal Cells (MSCs) Plus Standard of Care in Patients With Anthracycline-Associated Cardiomyopathy
This randomized pilot phase I trial studies the side effects of donor bone marrow derived mesenchymal stem cells in controlling heart failure in patients with cardiomyopathy caused by anthracyclines. Donor bone marrow derived mesenchymal stem cells may help to control symptoms of heart failure and improve heart function.
Read the detailed description
PRIMARY OBJECTIVE:
- To demonstrate the safety of allogeneic human mesenchymal stem cells (hMSCs) administered by intravenous infusion and transendocardial injection 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 and transendocardial injection 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 3 arms.
ARM I: Patients receive hMSCs intravenously (IV) over 10-20 minutes on days 1, 14, 21, and 28 and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity.
ARM II: Patients receive hMSCs transendocardially for a total of 15 injections and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity.
ARM III: Patients receive standard of care treatment for heart failure.
After completion of study treatment, patients are followed up periodically.
- Conditions
- Cardiomyopathy, Heart Failure, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
- Being tested
- Best Practice, Laboratory Biomarker Analysis, Mesenchymal Stem Cell Transplantation
- Medical subject headings
- Cardiomyopathies, Heart Failure, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Best PracticeOther
Also called standard of care, standard therapy
Given standard of care
Laboratory Biomarker AnalysisOther
Correlative studies
Mesenchymal Stem Cell TransplantationDrug
Given IV
Mesenchymal Stem Cell TransplantationDrug
Given transendocardially
| Study group | Role | What they receive |
|---|---|---|
| Arm I (hMSCs IV) | Experimental | Patients receive hMSCs IV over 10-20 minutes on days 1, 14, 21, and 28 and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity. Receives: Other: Best Practice; Other: Laboratory Biomarker Analysis; Drug: Mesenchymal Stem Cell Transplantation |
| Arm II (hMSCs transendocardially) | Experimental | Patients receive hMSCs transendocardially for a total of 15 injections and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity. Receives: Other: Best Practice; Other: Laboratory Biomarker Analysis; Drug: Mesenchymal Stem Cell Transplantation |
| Arm III (standard of care) | Active comparator | Patients receive standard of care treatment for heart failure. Receives: Other: Best Practice; Other: Laboratory Biomarker Analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Statistical analyses of safety will be descriptive.
Measured: Up to 6 months
Change in left ventricular ejection fraction (LVEF)
The comparison will be between the two groups of patients.
Measured: Baseline to 6 months
Secondary outcomes (7)
Change in improvement of left ventricular (LV) systolic function as assessed by LVEF
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Baseline up to 6 months
LV end-systolic and end-diastolic volumes as determined by contrast-enhanced 2-dimensional(D)/3D echography
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Re-hospitalization after 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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Aborted death from an automatic implantable cardioverter defibrillator (AICD) firing
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Nonfatal myocardial infarction
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Revascularization
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 each investigational group and the control group. If both investigation groups are significant at the p < .05 level, then the two investigational drugs can be compared using a gatekeeping procedure. These intervals and the associated p-values will be calculated using two-sample t-tests, with no adjustments for multiple comparisons.
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2016 (estimated)
- Study start18 Jul 2020
- Primary completion30 Jul 2026 (estimated)
- Study completion30 Jul 2026 (estimated)
Why it stopped: PI Request
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
- 72 participants (estimated)
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
- Paul Lin, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02962661
- Sponsor's protocol ID
- 2015-0835
- Other IDs
- NCI-2016-01921, 2015-0835
- Status last verified
- Jul 2026
- First submitted
- 8 Nov 2016
- Last updated
- 3 Aug 2026
- 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 3 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized 3-Arm Trial With Standard of Care Alone vs Either Intravenous Infusion or Transendocardial Injection of Allogeneic Bone Marrow Derived Multipotent Mesenchymal Stromal Cells (MSCs) Plus Standard of Care in Patients With Anthracycline-Associated Cardiomyopathy
- ClinicalTrials.gov ID
- NCT02962661
- Acronym / sponsor ID
- 2015-0835
- Conditions
- Cardiomyopathy, Heart Failure, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: PI Request
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 18 Jul 2020 (actual)
- Primary completion
- 30 Jul 2026 (estimated)
- Study completion
- 30 Jul 2026 (estimated)
- First posted
- 11 Nov 2016 (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 of donor bone marrow derived mesenchymal stem cells in controlling heart failure in patients with cardiomyopathy caused by anthracyclines. Donor bone marrow derived mesenchymal stem cells may help to control symptoms of heart failure and improve heart function.
Detailed description
PRIMARY OBJECTIVE:
- To demonstrate the safety of allogeneic human mesenchymal stem cells (hMSCs) administered by intravenous infusion and transendocardial injection 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 and transendocardial injection 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 3 arms.
ARM I: Patients receive hMSCs intravenously (IV) over 10-20 minutes on days 1, 14, 21, and 28 and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity.
ARM II: Patients receive hMSCs transendocardially for a total of 15 injections and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity.
ARM III: Patients receive standard of care treatment for heart failure.
After completion of study treatment, patients are followed up periodically.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm I (hMSCs IV) | Experimental | Other: Best Practice, Other: Laboratory Biomarker Analysis, Drug: Mesenchymal Stem Cell TransplantationPatients receive hMSCs IV over 10-20 minutes on days 1, 14, 21, and 28 and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity. |
| Arm II (hMSCs transendocardially) | Experimental | Other: Best Practice, Other: Laboratory Biomarker Analysis, Drug: Mesenchymal Stem Cell TransplantationPatients receive hMSCs transendocardially for a total of 15 injections and standard of care treatment for heart failure in the absence of disease progression or unacceptable toxicity. |
| Arm III (standard of care) | Active comparator | Other: Best Practice, Other: Laboratory Biomarker AnalysisPatients receive standard of care treatment 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 (7 listed)
- Patients with LVEF </= 40% documented from treatment with anthracyclines for any malignancy at any dose at any time without evidence of other causes of cardiomyopathy.
- Age >/= 18 and </= 90 years of age. English and non-English speaking patients are eligible.
- Documented NYHA class I, II and III.
- For patients who have received trastuzumab: Persistent LV dysfunction must be present 90 days after discontinuation of trastuzumab.
- Able to perform 6 minute walk test.
Key exclusion criteria (19 listed)
- Evidence of Ischemic Heart Disease as determined by study cardiologist.
- Significant Valvular Disease. (AS with AVA <1.5 and severe AR and MR)
- History of Familial Cardiomyopathy.
- Recent documented myocarditis within 2 months of enrollment.
- 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 |
|---|---|---|
| Best PracticeAlso: standard of care, standard therapyGiven standard of care | Other | Arm I (hMSCs IV), Arm II (hMSCs transendocardially), Arm III (standard of care) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (hMSCs IV), Arm II (hMSCs transendocardially), Arm III (standard of care) |
| Mesenchymal Stem Cell TransplantationGiven IV | Drug | Arm I (hMSCs IV) |
| Mesenchymal Stem Cell TransplantationGiven transendocardially | Drug | Arm II (hMSCs transendocardially) |
Primary outcome
Incidence of adverse events
Time frame: Up to 6 months
Statistical analyses of safety will be descriptive.
Change in left ventricular ejection fraction (LVEF)
Time frame: Baseline to 6 months
The comparison will be between the two groups of patients.
7 secondary outcomes
Change in improvement of left ventricular (LV) systolic function as assessed by LVEF
Time frame: Baseline up to 6 months
LV end-systolic and end-diastolic volumes as determined by contrast-enhanced 2-dimensional(D)/3D echography
Time frame: Up to 6 months
Cardiac death
Time frame: Up to 6 months
Re-hospitalization after heart failure
Time frame: Up to 6 months
Aborted death from an automatic implantable cardioverter defibrillator (AICD) firing
Time frame: Up to 6 months
Nonfatal myocardial infarction
Time frame: Up to 6 months
Revascularization
Time frame: Up to 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2026.
- MD Anderson Cancer CenterLink 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.
Paul Lin, MD 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 Best Practice, 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 7 inclusion and 19 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: paused, as of the sponsor's last update on 3 Aug 2026. 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?
Results are not available in the source record.
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 (time frame: Up to 6 months). 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 3 Aug 2026.