Dilated CardiomyopathyPhase 1NCT06464588
A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)
A Phase 1 study testing Allogeneic Neonatal mesenchymal stromal cells (nMSCs) in people with Dilated Cardiomyopathy, run by Emory University at 5 sites in United States.
Trial summary
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopathy. The purpose of the study is to help doctors and scientists learn if allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions are a safe and effective… Read more about this study
- Who is it for?
- Ages 4 to 40 years
- Conditions the study is for
- Dilated Cardiomyopathy
- What is being tested?
- Allogeneic Neonatal mesenchymal stromal cells (nMSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 5 sitesUnited States
- Study dates
- 14 Jul 2025to Jul 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 4 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Phase 1A: Age greater than or equal to 16 years and less than 40 years (≥16 years, <40 years).
- Phase 1B: Age greater than or equal to 4 years and less than 16 years (≥4 years, <16 years)
- Subjects must be able to sign their own consent for Phase 1A of the study.
- Diagnosis of dilated cardiomyopathy (DCM) defined as
- Any Congenital Cardiac Malformation with systemic ventricular systolic dysfunction; Idiopathic Cardiomyopathy; Familial/Inherited and/or Genetic Cardiomyopathy; History of Myocarditis; Acquired (Chemotherapy, Iatrogenic, Infection, Rheumatic, Nutritional); Ischemic (e.g. Kawasaki Disease, post-operative); Left ventricular noncompaction; Coronary Artery Disease
- Left ventricular ejection fraction less than or equal to 45% documented by two-dimensional echocardiogram or cardiac MRI within the prior six months.
Show all 10Show fewer
- Left ventricular dilation as defined by echocardiography left ventricular and end-diastolic dimension Z score > +2.0
- Biventricular physiology with systemic left ventricle
- Must receive guideline directed heart failure as defined by the American Heart Association, American College of Cardiology, and Heart Failure Society of America 118
- Have been unresponsive or poorly responsive to at least 3 months of maximum guideline directed treatments.
Who cannot join
- Listed for heart transplantation (as UNOS status 1A) or hospitalized while waiting for transplant (while on inotropes or with ventricular assist device)
- Cardiovascular surgery of percutaneous intervention to palliate or correct congenital cardiovascular malformations within 3 months of the screening visit. Patients anticipated to undergo corrective heart surgery during the 12 months after entry into Part 1A/1B.
- Previous heart transplant recipient
- Unoperated primary obstructive or severe regurgitant valve (aortic, pulmonary, mitral or tricuspid) disease, or significant systemic ventricular outflow obstruction or aortic arch obstruction anticipated to require surgical or transcatheter intervention within 6 months.
- Restrictive or hypertrophic cardiomyopathy
- Cardiogenic shock
Show all 32Show fewer
- Currently on extracorporeal membrane oxygenation support
- Ventricular assist device support
- Lethal, uncontrollable arrhythmia defined as an arrhythmia resulting in hemodynamic instability requiring need for defibrillation, continuous intravenous anti-arrhythmic medication or mechanical circulatory support
- Patients with persistent atrial fibrillation requiring specific pharmacotherapy
- Amyloidosis
- Ischemic dilated cardiomyopathy
- Clinical history of malignant neoplasm within 5 years (with the exception of curatively treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma)
- Serious neurologic disorder including loss of vision, stroke, or paralysis
- High-grade pulmonary embolism requiring interventional catheter procedure or pulmonary hypertension requiring use of pulmonary vasodilators including phosphodiesterase inhibitor or nitric oxide
- High-grade renal failure [eGFR<45] mL/min/1.73 m2 - serum potassium >5.3 mmol/L
- Multiple organ failure
- Non-cardiac condition that limits life span for <1 year
- Uncontrolled diabetes (HbA1c >9%) at screening
- Active infection (including endocarditis) requiring pharmacotherapy
- Sepsis
- Active hemorrhagic disease (e.g., gastrointestinal bleeding, injury)
- History of cardiac transplantation
- Immune system-altering medications, or immunosuppressive therapy at the time of enrolment or within the prior 12 weeks
- Dystrophin-associated cardiomyopathy confirmed by standard cardiomyopathy panel testing
- Confirmed myocarditis at time of screening
- Elevated LFTs greater than 2 times upper limit of normal at time of consent
- Elevated WBC greater than upper limit of normal as defined by local lab at time of consent
- Presence of HLA antibodies specific for therapeutic study product
- History of noncompliance, alcohol abuse, recreational drug use, or incarceration within the last year
- Currently pregnant or breastfeeding
- Unsafe/unfeasible to enroll due to PI/designee discretion
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Grady Memorial Hospital
Atlanta, Georgia, 30303
William Mahle, MD
William Mahle, MD
- Recruiting
Hughes Spalding Children's Hospital
Atlanta, Georgia, 30303
William Mahle, MD
William Mahle, MD
- Recruiting
Emory University Hospital
Atlanta, Georgia, 30322
William Mahle, MD
William Mahle, MD
- Recruiting
Arthur M. Blank Hospital
Atlanta, Georgia, 30329
William Mahle, MD
William Mahle, MD
- Recruiting
Scottish Rite Children's Hospital
Atlanta, Georgia, 30342
William Mahle, MD
William Mahle, MD
About this study
This is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopathy. The purpose of the study is to help doctors and scientists learn if allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions are a safe and effective way to improve cardiac function and left ventricular ejection fraction.
Read the detailed description
This study is a single site open label study with 2 phases. The 2 phases will be broken into an adult group Phase 1A with group pediatric group Phase 1B. This study will enroll patients between the ages of 4 years and 30 years old. The investigators will be looking at the safety, feasibility, and maximum tolerated dose of allogeneic neonatal mesenchymal stromal cells (nMSCs) as defined by freedom from CTCAE or Grade 3 AE that is probably or definitely related to the IP throughout the duration of the study.
A minimum of 9 and a maximum of 18 patients will be enrolled into both Phase 1A adult groups and 1B pediatric groups. Phase 1A subjects will receive study products with doses defined by the study group and Phase 1B will begin after all adult subjects have completed Phase 1A infusions, FDA and a DSMC review. Phase 1B subjects will receive study product dosed per body weight in the defined study group.
Allogeneic neonatal mesenchymal stromal cells (nMSCs) will be infused via IV every 30 days for a total of 3 infusions. There will be a baseline visit before allogeneic neonatal mesenchymal stromal cells (nMSCs) therapy is initiated, followed by a phone call 30 days after the last infusion. There will be in person visits at 3-month, 6 months, and 1 year mark. The total duration for each patient will be 14 months.
Labs will be collected at baseline, during nMSC infusions, and at in person follow up visits to assess cardiac function. Any leftover blood samples may be stored for future research by the sponsor of the study. Echocardiograms will be completed at baseline, and 3 month-, 6 month-, and 1-year visits to look at left ventricular ejection fraction and electrocardiograms will be completed to provide measures of cardiac rhythm or rhythm. Other assessments include physical exam, 6-minute walk test, Cardiac MRI, vital signs, PedsQL questionnaire for pediatrics and The Kansas City Cardiomyopathy Questionnaire for adults.
It is expected to recruit 18-36 participants through face-to-face encounters between participants and study staff during clinical encounters at Children's Healthcare of Atlanta, Emory Health care system, and Grady Health, Clinicaltrials.gov registration, and Institutional Review Board approved advertisements to surrounding hospitals with cardiac programs. Patients will also be recruited through MyChart. If identified as eligible to participate, the study team will seek approval by the subjects' primary cardiologist and consent and/or assent with the permission of the parent or legally authorized representative, will occur in person during a baseline visit.
After allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions, patients will not be required to stay overnight for a follow-up visit. There will be financial compensation for each study visit, and patients will be reimbursed for parking.
- Conditions
- Dilated Cardiomyopathy
- Being tested
- Allogeneic Neonatal mesenchymal stromal cells (nMSCs)
- Keywords
- Dilated Cardiomyopathy, Neonatal Mesenchymal Cells (nMSCs)
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Neonatal mesenchymal stromal cells (nMSCs)Biological
Also called nMSCs infusions
nMSCs will be administered intravenously in the predefined dose per each group. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels.
| Study group | Role | What they receive |
|---|---|---|
| Adult Cohort | Experimental | Adults (≥16 years to <40 years) will be enrolled into all dose levels (as tolerated) of Phase 1A. Open label nMSCs will be administered intravenously in the following defined dose groups. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels:
* Dose level 1: 5.0x107 nMSCs at 0, 15 and 30 days
* Dose level 2: 1.0.x108 nMSCs at 0, 15 and 30 days
* Dose level 3: 2.5x108 nMSCs at 0, 15 and 30 days
Dose escalation will follow 3+3 study design parameters. The treatment between the first and second patients of each dose level will be staggered at least one month after the first patient's first infusion within each dose level. The next dosing group will be initiated at least one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation. Receives: Biological: Allogeneic Neonatal mesenchymal stromal cells (nMSCs) |
| Pediatric Cohort | Experimental | Pediatric participants (4 to ≤16 years) will be enrolled as determined in Phase 1B(3+3 study design; open label). nMSCs will be administered intravenously (IV) in the following defined dose groups. The rate of infusion will be approx. 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels:
* Dose Level 1: 0.7x106 nMSCs/kg
* Dose Level 2: 1.43x106 nMSCs/kg
* Dose Level 3: 2.85x106 nMSCs/kg
Dose escalation will follow 3+3 study design parameters. Treatment between 1st and 2nd patients of each dose level will be staggered at least 1 month after the 1st patient's first infusion within each dose level. The next dosing group will be initiated one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation. Following IV delivery of nMSCs, patients will be followed at 3m, 6m and 1yr. Receives: Biological: Allogeneic Neonatal mesenchymal stromal cells (nMSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater AE that is probably or related to the IP throughout the duration of the study will be recorded. Results can vary from 0 to 100% and higher proportion correlates with better outcome.
TCAE Grade 3 is defined as severe or medically significant not immediately life-threatening; hospitalization or prolongation of hospitalization.
Grade 4 and 5 AEs include composite of: death, life-threatening events, initial or prolonged hospitalization, disability of permanent damage and congenital anomaly/birth defects.
Measured: End of study, around 12 months post-intervention
Maximum tolerated dose (MTD) in patients with dilated cardiomyopathy
If two patients in a dosing group have a related SAE or Dose Limiting Toxicity (DLT), then the previous dosing group will be defined as MTD.
Measured: End of study, around 12 months post-intervention
Secondary outcomes (5)
Change of left ventricular ejection fraction (LVEF) from baseline
Change of left ventricular ejection fraction (LVEF) between baseline, 3-month, 6-month, and 12-month follow-up determined by echocardiography and cardiac MRI.
Measured: Baseline, 3-month, 6-month, and 12-month post-intervention
Change in N-terminal pro b-type natriuretic peptide (NT-proBNP) levels from baseline
N-terminal pro b-type natriuretic peptide (NT-proBNP) levels will be collected on infusion days, and all post infusion follow up visits.
Measured: Baseline, 3-month, 6-month, and 12-month post-intervention
Change of 6-minute walk test (6MWT) results
A 6-minute walk test (6MWT) will be completed at baseline, 3-month, 6-month, and 1-year visits to measure functional status if the participant is developmentally appropriate. Distance in meters will be measured until the participant can either walk 6 minutes, or they become too exhausted.
Measured: Baseline, 3-month, 6-month, and 12-months post-intervention
Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey
Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey (≥4 years - < 18 years). Total possible score ranges from 0 to 100 and higher scores indicate better QOL.
Measured: Baseline, 3-month, 6-month and 12-month post-intervention
Change in Kansas City Cardiomyopathy Questionnaire
Change in Kansas City Cardiomyopathy Questionnaire will be recorded (≥18 years - ≤30 years). KCCQ scores are scaled from 0 to 100 and summarized in 25-point ranges, where scores represent health status as follows: 0 to 24: very poor to poor; 25 to 49: poor to fair; 50 to 74: fair to good; and 75 to 100: good to excellent.
Measured: Baseline, 3-month, 6-month and 12-month post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2024
- Study start14 Jul 2025
- Primary completionJul 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated CardiomyopathyStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation
- Responsible party
- Principal investigator, William T. Mahle, Emory University
- Investigators
- William Mahle, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06464588
- Sponsor's protocol ID
- STUDY00004268
- Other IDs
- 2025P013052
- Status last verified
- Jun 2026
- First submitted
- 13 Jun 2024
- Last updated
- 26 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after deidentification and publication. Data will be shared with Investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose, to achieve aims in the approved proposal.
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)
- ClinicalTrials.gov ID
- NCT06464588
- Acronym / sponsor ID
- STUDY00004268
- Conditions
- Dilated Cardiomyopathy
- Keywords
- Dilated Cardiomyopathy, Neonatal Mesenchymal Cells (nMSCs)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- The Marcus Foundation
- Study start
- 14 Jul 2025 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 18 Jun 2024 (actual)
- 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 is a Phase 1 study to determine the safety and efficacy of allogeneic neonatal mesenchymal stromal cells (nMSCs) for the treatment of Dilated Cardiomyopathy. The purpose of the study is to help doctors and scientists learn if allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions are a safe and effective way to improve cardiac function and left ventricular ejection fraction.
Detailed description
This study is a single site open label study with 2 phases. The 2 phases will be broken into an adult group Phase 1A with group pediatric group Phase 1B. This study will enroll patients between the ages of 4 years and 30 years old. The investigators will be looking at the safety, feasibility, and maximum tolerated dose of allogeneic neonatal mesenchymal stromal cells (nMSCs) as defined by freedom from CTCAE or Grade 3 AE that is probably or definitely related to the IP throughout the duration of the study.
A minimum of 9 and a maximum of 18 patients will be enrolled into both Phase 1A adult groups and 1B pediatric groups. Phase 1A subjects will receive study products with doses defined by the study group and Phase 1B will begin after all adult subjects have completed Phase 1A infusions, FDA and a DSMC review. Phase 1B subjects will receive study product dosed per body weight in the defined study group.
Allogeneic neonatal mesenchymal stromal cells (nMSCs) will be infused via IV every 30 days for a total of 3 infusions. There will be a baseline visit before allogeneic neonatal mesenchymal stromal cells (nMSCs) therapy is initiated, followed by a phone call 30 days after the last infusion. There will be in person visits at 3-month, 6 months, and 1 year mark. The total duration for each patient will be 14 months.
Labs will be collected at baseline, during nMSC infusions, and at in person follow up visits to assess cardiac function. Any leftover blood samples may be stored for future research by the sponsor of the study. Echocardiograms will be completed at baseline, and 3 month-, 6 month-, and 1-year visits to look at left ventricular ejection fraction and electrocardiograms will be completed to provide measures of cardiac rhythm or rhythm. Other assessments include physical exam, 6-minute walk test, Cardiac MRI, vital signs, PedsQL questionnaire for pediatrics and The Kansas City Cardiomyopathy Questionnaire for adults.
It is expected to recruit 18-36 participants through face-to-face encounters between participants and study staff during clinical encounters at Children's Healthcare of Atlanta, Emory Health care system, and Grady Health, Clinicaltrials.gov registration, and Institutional Review Board approved advertisements to surrounding hospitals with cardiac programs. Patients will also be recruited through MyChart. If identified as eligible to participate, the study team will seek approval by the subjects' primary cardiologist and consent and/or assent with the permission of the parent or legally authorized representative, will occur in person during a baseline visit.
After allogeneic neonatal mesenchymal stromal cells (nMSCs) infusions, patients will not be required to stay overnight for a follow-up visit. There will be financial compensation for each study visit, and patients will be reimbursed for parking.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Adult Cohort | Experimental | Biological: Allogeneic Neonatal mesenchymal stromal cells (nMSCs)Adults (≥16 years to <40 years) will be enrolled into all dose levels (as tolerated) of Phase 1A. Open label nMSCs will be administered intravenously in the following defined dose groups. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels: * Dose level 1: 5.0x107 nMSCs at 0, 15 and 30 days * Dose level 2: 1.0.x108 nMSCs at 0, 15 and 30 days * Dose level 3: 2.5x108 nMSCs at 0, 15 and 30 days Dose escalation will follow 3+3 study design parameters. The treatment between the first and second patients of each dose level will be staggered at least one month after the first patient's first infusion within each dose level. The next dosing group will be initiated at least one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation. |
| Pediatric Cohort | Experimental | Biological: Allogeneic Neonatal mesenchymal stromal cells (nMSCs)Pediatric participants (4 to ≤16 years) will be enrolled as determined in Phase 1B(3+3 study design; open label). nMSCs will be administered intravenously (IV) in the following defined dose groups. The rate of infusion will be approx. 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels: * Dose Level 1: 0.7x106 nMSCs/kg * Dose Level 2: 1.43x106 nMSCs/kg * Dose Level 3: 2.85x106 nMSCs/kg Dose escalation will follow 3+3 study design parameters. Treatment between 1st and 2nd patients of each dose level will be staggered at least 1 month after the 1st patient's first infusion within each dose level. The next dosing group will be initiated one month after the last subject in a particular dose level has received the last dose treatment. Once MTD has been determined, 3 additional patients will be enrolled to ensure a total of 6 patients are enrolled in MTD level for confirmation. Following IV delivery of nMSCs, patients will be followed at 3m, 6m and 1yr. |
Who may qualify
- Ages
- 4 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Phase 1A: Age greater than or equal to 16 years and less than 40 years (≥16 years, <40 years).
- Phase 1B: Age greater than or equal to 4 years and less than 16 years (≥4 years, <16 years)
- Subjects must be able to sign their own consent for Phase 1A of the study.
- Diagnosis of dilated cardiomyopathy (DCM) defined as
- Must receive guideline directed heart failure as defined by the American Heart Association, American College of Cardiology, and Heart Failure Society of America 118
Key exclusion criteria (32 listed)
- Listed for heart transplantation (as UNOS status 1A) or hospitalized while waiting for transplant (while on inotropes or with ventricular assist device)
- Cardiovascular surgery of percutaneous intervention to palliate or correct congenital cardiovascular malformations within 3 months of the screening visit. Patients anticipated to undergo corrective heart surgery during the 12 months after entry into Part 1A/1B.
- Previous heart transplant recipient
- Unoperated primary obstructive or severe regurgitant valve (aortic, pulmonary, mitral or tricuspid) disease, or significant systemic ventricular outflow obstruction or aortic arch obstruction anticipated to require surgical or transcatheter intervention within 6 months.
- Restrictive or hypertrophic 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 |
|---|---|---|
| Allogeneic Neonatal mesenchymal stromal cells (nMSCs)Also: nMSCs infusionsnMSCs will be administered intravenously in the predefined dose per each group. The rate of infusion will be approximately 30- 60 minutes at 0, 15 and 30 days, with escalating dose levels. | Biological | Adult Cohort, Pediatric Cohort |
Primary outcome
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater
Time frame: End of study, around 12 months post-intervention
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater AE that is probably or related to the IP throughout the duration of the study will be recorded. Results can vary from 0 to 100% and higher proportion correlates with better outcome. TCAE Grade 3 is defined as severe or medically significant not immediately life-threatening; hospitalization or prolongation of hospitalization. Grade 4 and 5 AEs include composite of: death, life-threatening events, initial or prolonged hospitalization, disability of permanent damage and congenital anomaly/birth defects.
Maximum tolerated dose (MTD) in patients with dilated cardiomyopathy
Time frame: End of study, around 12 months post-intervention
If two patients in a dosing group have a related SAE or Dose Limiting Toxicity (DLT), then the previous dosing group will be defined as MTD.
5 secondary outcomes
Change of left ventricular ejection fraction (LVEF) from baseline
Time frame: Baseline, 3-month, 6-month, and 12-month post-intervention
Change in N-terminal pro b-type natriuretic peptide (NT-proBNP) levels from baseline
Time frame: Baseline, 3-month, 6-month, and 12-month post-intervention
Change of 6-minute walk test (6MWT) results
Time frame: Baseline, 3-month, 6-month, and 12-months post-intervention
Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey
Time frame: Baseline, 3-month, 6-month and 12-month post-intervention
Change in Kansas City Cardiomyopathy Questionnaire
Time frame: Baseline, 3-month, 6-month and 12-month post-intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2026.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after deidentification and publication. Data will be shared with Investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose, to achieve aims in the approved proposal.
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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.
William Mahle, MD principal investigator
Emory University
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Allogeneic Neonatal mesenchymal stromal cells (nMSCs) in people with Dilated Cardiomyopathy, run by Emory University at 5 sites 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 10 inclusion and 32 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: looking for participants now, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact William Mahle, MD (404-256-2593, wmahle@emory.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater (time frame: End of study, around 12 months post-intervention). 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 26 Jun 2026.