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

Primary Purpose

Congenital Heart Disease (CHD)

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
BM-MSC
Sponsored by
Catherine Bollard
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Heart Disease (CHD)

Eligibility Criteria

undefined - 6 Months (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's National Health System

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

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

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of subjects who experience serious adverse events, adverse events, and/or early treatment discontinuations.
Dose Limiting Toxicity is attributable to the MSC administration.

Secondary Outcome Measures

Actual magnitude of differences in neuroimaging and neurodevelopmental variables will be measured after MSC delivery.
Secondary objective will be measured by using the Pediatric Cardiac Critical Care Consortium (PC4) registry system.

Full Information

First Posted
January 15, 2020
Last Updated
September 19, 2023
Sponsor
Catherine Bollard
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1. Study Identification

Unique Protocol Identification Number
NCT04236479
Brief Title
Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)
Official Title
Mesenchymal Stromal Cells Delivery Through Cardiopulmonary Bypass in Pediatric Cardiac Surgery
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 29, 2020 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Catherine Bollard

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Heart Disease (CHD)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Bone marrow-derived mesenchymal stromal cell (BM-MSC)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
BM-MSC
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of subjects who experience serious adverse events, adverse events, and/or early treatment discontinuations.
Description
Dose Limiting Toxicity is attributable to the MSC administration.
Time Frame
45 days following the MSC administration
Secondary Outcome Measure Information:
Title
Actual magnitude of differences in neuroimaging and neurodevelopmental variables will be measured after MSC delivery.
Description
Secondary objective will be measured by using the Pediatric Cardiac Critical Care Consortium (PC4) registry system.
Time Frame
18 months

10. Eligibility

Sex
All
Maximum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Richard Jonas, MD
Organizational Affiliation
CNMC
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's National Health System
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States

12. IPD Sharing Statement

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

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