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Stem Cell Therapy

Neonatal Mesenchymal Cells for Dilated Cardiomyopathy

Phase 1

Waitlist Available

Led By William Mahle, MD

Research Sponsored by Emory University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects must be able to sign their own consent for Phase 1A of the study

Diagnosis of dilated cardiomyopathy (DCM) as defined by specific criteria including left ventricular ejection fraction ≤45%, left ventricular dilation, biventricular physiology, unresponsiveness to guideline-directed treatments

Must not have

Listed for heart transplantation (UNOS status 1A) or hospitalized while waiting for transplant

Specific cardiac conditions including severe valve disease, restrictive or hypertrophic cardiomyopathy, cardiogenic shock

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 3-month, 6-month and 12-month post-intervention

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to test if using special cells from newborns can help improve heart function in patients with Dilated Cardiomyopathy. The study is looking to see if these cells are safe and effective

Who is the study for?

This trial is for young adults and children with Dilated Cardiomyopathy (DCM), a condition where the heart becomes weakened and enlarged. Participants must meet certain health criteria to be included, but specific inclusion details are not provided here.

What is being tested?

The study tests if infusions of allogeneic neonatal mesenchymal stromal cells (nMSCs) can safely improve heart function in patients with DCM. It's an early-stage trial to assess safety and how well the treatment works.

What are the potential side effects?

Since this is a Phase 1 trial primarily focused on safety, potential side effects are being studied; however, common risks may include immune reactions or infusion-related responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can sign my own consent for the study.

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I have been diagnosed with DCM and my heart doesn’t pump well.

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I am between 18 and 29 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am on the heart transplant list as a top priority or hospitalized waiting for it.

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I do not have severe heart valve disease or specific heart muscle conditions.

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I have not had recent serious cancer, brain disorders, severe lung clots, high blood pressure, or kidney failure.

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I have had heart surgery or treatment for heart defects recently.

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I have a history of heart transplant or specific heart conditions.

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I have a history of serious heart rhythm problems.

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I do not have uncontrolled diabetes, active infections, sepsis, or bleeding disorders.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 3-month, 6-month and 12-month post-intervention

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 3-month, 6-month and 12-month post-intervention

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 3-month, 6-month and 12-month post-intervention for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Maximum tolerated dose (MTD) in patients with dilated cardiomyopathy

Proportion of participants with freedom from any Common Toxicity Criteria for Adverse Events (CTCAE) Grade 3 or greater

Secondary study objectives

Change in Kansas City Cardiomyopathy Questionnaire

Change in N-terminal pro b-type natriuretic peptide (NT-proBNP) levels from baseline

Change in quality-of-life validated survey scores in Peds Quality of Life (QOL) survey

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Pediatric CohortExperimental Treatment1 Intervention

Pediatric participants (ages 4 years-18 years) will be enrolled into dose level as determined in Phase 1B (3+3 study design; 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: 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. 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. Following intravenous delivery of nMSCs, patients will be followed at 3 months, 6 months and 1 year from the last infusion to complete all safety and efficacy assessments.

Group II: Adult CohortExperimental Treatment1 Intervention

Adults (18-30 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 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.

0 / 10Eligibility criteria met