Neonatal Mesenchymal Cells for Dilated Cardiomyopathy

Recruiting in Palo Alto (17 mi)

+4 other locations

Overseen ByWilliam Mahle, MD

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Emory University

No Placebo Group

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

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.

Inclusion Criteria

I can sign my own consent for the study.

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

I am between 18 and 29 years old.

Exclusion Criteria

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

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

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

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

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

I have a history of serious heart rhythm problems.

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

Participant Groups

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.

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.