NCT06464588

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)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Emory University · Academic / Other
• Sex: All
• Age: 4 Years – 30 Years
• Healthy volunteers: Not accepted

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 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. All pediatric 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. A check-in will be scheduled the morning after each infusion for pediatric patients. 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 and Emory Health care system, 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, pediatric patients will be provided an overnight stay at Ronald McDonald House, or a hospital affiliated hotel in case of any unanticipated effects and follow up visits the next morning. This is so pediatric patients are near the hospital for such events. Adults 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

Interventions

Type	Name	Description
BIOLOGICAL	Allogeneic Neonatal mesenchymal stromal cells (nMSCs)	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.

Timeline

• Start date: 2025-07-14
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2024-06-18
• Last updated: 2025-09-10

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study