Clinical Trial Details
— Status: Active, not recruiting
Administrative data
NCT number |
NCT04236479 |
Other study ID # |
Pro00011914 |
Secondary ID |
|
Status |
Active, not recruiting |
Phase |
Phase 1
|
First received |
|
Last updated |
|
Start date |
July 29, 2020 |
Est. completion date |
April 2025 |
Study information
Verified date |
September 2023 |
Source |
Children's National Research Institute |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
The proposed study will be a prospective, open-label, single-center, safety and feasibility
phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery
though cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital
heart disease (CHD) who will be undergoing a two-ventricle repair within the first six months
of life
Description:
This study is a prospective, open-label, single-center, safety and feasibility phase 1 trial
of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though
cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital heart
disease (CHD) who will be undergoing a two-ventricle repair within the first six months of
life. The dose-escalation methods with a modified continual reassessment at the five dose
levels (1x10^6, 10x10^6, 20x10^6, 40x10^6, 80x10^6, cells/kg) will be performed to determine
safety and feasibility of allogeneic BM-MSC infusion during pediatric cardiac surgery and the
maximum tolerated dose in infants with CHD. In addition to the primary objective of assessing
the safety and feasibility of BM-MSC delivery through CPB, our secondary objectives are
designed to develop biological signature measures and clinical outcome measures feasible for
use in larger efficacy and effectiveness trials with a particular focus on neurodevelopmental
outcome and early postoperative course after BM-MSC treatment. We will determine actual
magnitude of differences in neuroimaging and neurodevelopmental variables and postoperative
inflammatory and pathophysiological variables after BM-MSC delivery in infants with CHD.
Enrollment, follow-up, and analysis are planned to occur over 36 months for the treatment and
initial follow-up portions of the study. Long-term follow-up until 18 months of age will be
subsequently reported.