Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)
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ClinicalTrials.gov Identifier: NCT04236479 |
Recruitment Status :
Active, not recruiting
First Posted : January 22, 2020
Last Update Posted : September 21, 2023
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Condition or disease | Intervention/treatment | Phase |
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Congenital Heart Disease (CHD) | Biological: BM-MSC | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 17 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Mesenchymal Stromal Cells Delivery Through Cardiopulmonary Bypass in Pediatric Cardiac Surgery |
Actual Study Start Date : | July 29, 2020 |
Estimated Primary Completion Date : | September 2024 |
Estimated Study Completion Date : | April 2025 |
Arm | Intervention/treatment |
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Experimental: 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.
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Biological: 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. |
- Number of subjects who experience serious adverse events, adverse events, and/or early treatment discontinuations. [ Time Frame: 45 days following the MSC administration ]Dose Limiting Toxicity is attributable to the MSC administration.
- Actual magnitude of differences in neuroimaging and neurodevelopmental variables will be measured after MSC delivery. [ Time Frame: 18 months ]Secondary objective will be measured by using the Pediatric Cardiac Critical Care Consortium (PC4) registry system.
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Ages Eligible for Study: | up to 6 Months (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Neonatal and young infantile patients who are ≤ 3 months of age
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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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04236479
United States, District of Columbia | |
Children's National Health System | |
Washington, District of Columbia, United States, 20010 |
Principal Investigator: | Richard Jonas, MD | CNMC |
Responsible Party: | Catherine Bollard, Director, Center for Cancer and Immunology/ Center for Emerging Technologies in Immune Cell Therapies (CETI), Children's National Research Institute |
ClinicalTrials.gov Identifier: | NCT04236479 |
Other Study ID Numbers: |
Pro00011914 |
First Posted: | January 22, 2020 Key Record Dates |
Last Update Posted: | September 21, 2023 |
Last Verified: | September 2023 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Heart Diseases Heart Defects, Congenital Cardiovascular Diseases Cardiovascular Abnormalities Congenital Abnormalities |