## Protocol Section
### Identification Module
**NCT ID:** 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

#### Organization Study ID Info
**ID:** Pro00011914
#### Organization
**Class:** OTHER
**Full Name:** Children's National Research Institute
### Status Module
#### Completion Date
**Date:** 2025-04

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-09-21

**Type:** ACTUAL

**Last Update Submit Date:** 2023-09-19

**Overall Status:** ACTIVE_NOT_RECRUITING

#### Primary Completion Date
**Date:** 2024-09

**Type:** ESTIMATED

#### Start Date
**Date:** 2020-07-29

**Type:** ACTUAL

**Status Verified Date:** 2023-09

#### Study First Post Date
**Date:** 2020-01-22
**Type:** ACTUAL
**Study First Submit Date:** 2020-01-15
**Study First Submit QC Date:** 2020-01-17
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** Catherine Bollard

#### Responsible Party

**Investigator Affiliation:** Children's National Research Institute

**Investigator Full Name:** Catherine Bollard

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

**Type:** SPONSOR_INVESTIGATOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**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 Module

**Conditions:**

- Congenital Heart Disease (CHD)

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 17

**Type:** ACTUAL

**Phases:**

- PHASE1

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**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 Names:**

- Biological: BM-MSC

**Label:** Bone marrow-derived mesenchymal stromal cell (BM-MSC)

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

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

**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.

**Name:** BM-MSC

**Type:** BIOLOGICAL

### Outcomes Module

#### Primary Outcomes

**Description:** Dose Limiting Toxicity is attributable to the MSC administration.

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

**Time Frame:** 45 days following the MSC administration

#### Secondary Outcomes

**Description:** Secondary objective will be measured by using the Pediatric Cardiac Critical Care Consortium (PC4) registry system.

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

**Time Frame:** 18 months

### Eligibility Module

**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 Age:** 6 Months

**Sex:** ALL

**Standard Ages:**

- CHILD

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Washington

**Country:** United States

**Facility:** Children's National Health System

**State:** District of Columbia

**Zip:** 20010

#### Overall Officials

**Official 1:**

**Affiliation:** CNMC

**Name:** Richard Jonas, MD

**Role:** PRINCIPAL_INVESTIGATOR

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000002318

- Term: Cardiovascular Diseases

- ID: D000018376

- Term: Cardiovascular Abnormalities

- ID: D000000013

- Term: Congenital Abnormalities

### Condition Browse Module - Browse Branches

- Abbrev: BC14

- Name: Heart and Blood Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

### Condition Browse Module - Browse Leaves

- ID: M9419

- Name: Heart Diseases

- Relevance: HIGH

- As Found: Heart Disease

- ID: M9418

- Name: Heart Defects, Congenital

- Relevance: HIGH

- As Found: Congenital Heart Disease

- ID: M12

- Name: Congenital Abnormalities

- Relevance: LOW

- As Found: Unknown

- ID: M20503

- Name: Cardiovascular Abnormalities

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000006331

- Term: Heart Diseases

- ID: D000006330

- Term: Heart Defects, Congenital

### Misc Info Module

- Version Holder: 2024-05-24
