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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04236479
Recruitment Status : Not yet recruiting
First Posted : January 22, 2020
Last Update Posted : January 22, 2020
Sponsor:
Information provided by (Responsible Party):
Catherine Bollard, Children's National Research Institute

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 three months of life

Condition or disease Intervention/treatment Phase
Congenital Heart Disease (CHD) Biological: BM-MSC Phase 1

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 three 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
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
Estimated Study Start Date : February 2020
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Diseases

Arm Intervention/treatment
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.
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 three months of life.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 3 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Contacts
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Contact: Nobuyuki Ishibashi, MD 202-476-2388 NIshibas@childrensnational.org
Contact: Fahmida Hoq, MBBS, MS 202-476-3634 fhoq@childrensnational.org

Locations
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United States, District of Columbia
Children's National Health System
Washington, District of Columbia, United States, 20010
Sponsors and Collaborators
Catherine Bollard
Investigators
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Principal Investigator: Richard Jonas, MD CNMC

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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: January 22, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Heart Diseases
Heart Defects, Congenital
Cardiovascular Diseases
Cardiovascular Abnormalities
Congenital Abnormalities