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

NCT04236479 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: Pro00011914
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

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

Conditions

Congenital Heart Disease (CHD)

Outcome Measures

Primary Outcomes (1)

• Number of subjects who experience serious adverse events, adverse events, and/or early treatment discontinuations.

  Dose Limiting Toxicity is attributable to the MSC administration.

  45 days following the MSC administration

Secondary Outcomes (1)

• Actual magnitude of differences in neuroimaging and neurodevelopmental variables will be measured after MSC delivery.

  18 months

Study Arms (1)

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

EXPERIMENTAL

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.

Biological: BM-MSC

Interventions

BM-MSC 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.

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

Eligibility Criteria

• Age: Up to 6 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Catherine Bollard: lead

Study Sites (1)

Children's National Health System

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Heart Defects, Congenital

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Richard Jonas, MD

  CNMC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director, Center for Cancer and Immunology/ Center for Emerging Technologies in Immune Cell Therapies (CETI)

Study Record Dates

• First Submitted: January 15, 2020
• First Posted: January 22, 2020
• Study Start: July 29, 2020
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: April 29, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)