Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)

NCT ID: NCT04236479

Last Updated: 2025-04-29

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-07-29
• Study Completion Date: 2026-09-30

Brief 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

Detailed 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.

Conditions

Congenital Heart Disease (CHD)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Bone marrow-derived mesenchymal stromal cell (BM-MSC)

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.

Group Type: EXPERIMENTAL

BM-MSC

Intervention Type: BIOLOGICAL

Allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery through 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.

Interventions

BM-MSC

Allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery through 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.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Neonatal and young infantile patients who are ≤ 3 months of age
• Scheduled to undergo reparative two-ventricle repair for congenital heart defects without aortic arch reconstruction, including the following:

a. D-Transposition of the Great Arteries (d-TGA) Group: i. d-TGA with intact ventricular septum (d-TGA, IVS) ii. d-TGA with ventricular septal defect (d-TGA, VSD) b. Ventricular Septal Defect (VSD) Group: i. VSD without aortic arch obstruction (AAO) ii. Complete common atrioventricular canal defect (CAVC) c. Tetralogy of Fallot (TOF) Group: i. Tetralogy of Fallot (TOF) ii. Tetralogy of Fallot with Pulmonary Atresia (TOF,PA) iii. Truncus arteriosus (TA) iv. Double outlet right ventricle (DORV)

• Scheduled surgery at or before three months of age.
• Parent/guardian capable of providing informed consent.

Exclusion Criteria

• Birth weight less than 2.0 kg
• Recognizable phenotypic syndrome
• Associated extracardiac anomalies of greater than minor severity
• Previous cardiac surgery
• Associated cardiovascular anomalies requiring aortic arch reconstruction and/or additional open cardiac surgical procedures in infancy
• Prior severe hypoxic event
• Significant screening test values that place subjects at increased risk of complications from participation in the study

• Maximum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Catherine Bollard

OTHER

Sponsor Role: lead

Responsible Party

Catherine Bollard

Director, Center for Cancer and Immunology/ Center for Emerging Technologies in Immune Cell Therapies (CETI)

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Richard Jonas, MD

Role: PRINCIPAL_INVESTIGATOR

CNMC

Locations

Children's National Health System

Washington D.C., District of Columbia, United States

Countries

United States

Other Identifiers

Pro00011914

Identifier Type: -

Identifier Source: org_study_id