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)

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Emory University
Study ID
NCT06464588
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
4 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • Allogeneic Neonatal mesenchymal stromal cells (nMSCs) — BIOLOGICAL
    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 Details

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.

Key Dates

First listed
Jun 18, 2024
Start date
Jul 14, 2025
Status verified
Jun 2026
Primary completion
Jul 31, 2027
Completion
Jul 31, 2027

Study Design

Enrollment
36 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Adult Cohort
    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.
  • Experimental: Pediatric Cohort
    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.

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 ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Arthur M. Blank HospitalAtlantaGeorgia30329
William Mahle, MD
William Mahle, MD (PRINCIPAL_INVESTIGATOR)
Emory University HospitalAtlantaGeorgia30322
William Mahle, MD
William Mahle, MD (PRINCIPAL_INVESTIGATOR)
Grady Memorial HospitalAtlantaGeorgia30303
William Mahle, MD
William Mahle, MD (PRINCIPAL_INVESTIGATOR)
Hughes Spalding Children's HospitalAtlantaGeorgia30303
William Mahle, MD
William Mahle, MD (PRINCIPAL_INVESTIGATOR)
Scottish Rite Children's HospitalAtlantaGeorgia30342
William Mahle, MD
William Mahle, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Atlanta, GA

By research site
Arthur M. Blank Hospital· Atlanta, GAEmory University Hospital· Atlanta, GAGrady Memorial Hospital· Atlanta, GAHughes Spalding Children's Hospital· Atlanta, GAScottish Rite Children's Hospital· Atlanta, GA

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