Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD

NCT ID: NCT04907526

Last Updated: 2025-06-27

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-01
• Study Completion Date: 2025-08-30

Brief Summary

Researchers want to better understand what happens to the heart when the autologous (from one's own body) stem cells are injected directly into muscle of the right side of the heart during the Fontan (Stage III) surgery. They want to see if there are changes in the electrical activity, the structure, and the function of the heart following this stem cell-based therapy. Researchers will compare the results from people who receive the stem cells to the results from people who do not receive the stem cells.

Detailed Description

This Phase I study is a multicenter, prospective, open-label, non-randomized study designed to evaluate the safety, of autologous UCB-MNC delivered into the right ventricular myocardium of subjects with severe CHD defined by single right ventricular dependent circulatory systems at the time of a planned Fontan surgical palliation. This will be achieved by comparing the data collected in the treatment arm to the equivalent data collected in the control arm. The purpose of this non-randomized open-label Phase I clinical study is to prospectively evaluate the safety, as measured by the short-term and long-term safety endpoints and change in baseline comparatives, for the autologous UCB-MNC intramyocardial injections into the single, morphologically right ventricle of subjects with severe CHD requiring Fontan surgical palliation. The treatment group will be compared to an untreated control group.

Conditions

Congenital Heart Disease, SRV Dependent

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Arm

Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 3 million cells per kilogram of body weight. This is a one time treatment at the time of Stage III Fontan surgery.

Group Type: EXPERIMENTAL

Autologous mononuclear cells

Intervention Type: BIOLOGICAL

Autologous mononuclear cells delivered into right ventricle at time of Stage III Fontan surgery.

Control Arm

Control cohort not receiving the cell product, which will be enrolled and followed using the same inclusion/exclusion criteria and follow-up requirements as the treatment arm.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Autologous mononuclear cells

Autologous mononuclear cells delivered into right ventricle at time of Stage III Fontan surgery.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Diagnosis of a congenital hear defect with functionally single right ventricle (such as HLHS, HLHS variants, unbalanced AV septal defect with R dominance, DORV with Hypoplastic LV) undergoing Fontan surgical palliation
• At least 2 and less than or equal to 5 years of age at time of Fontan surgical palliation
• For subjects enrolling in the treatment arm, previous participation in clinical trial Umbilical Cord Blood Collection and Processing for Hypoplastic Left Heart Syndrome patients (NCT01856049) with autologous UCB-MNC product collected and available for distribution is required.

Exclusion Criteria

• History of DMSO reaction (treatment arm only subjects).
• Parent(s) or legal guardian unwilling to have their child participate or unwilling to follow the study procedures.
• Severe chronic diseases at the discretion of the treating physician.
• Extensive extra-cardiac syndromic features.
• History of cancer.
• Any of the following complications of his/her congenital heart disease:

• any condition requiring urgent, or unplanned intervention procedure within 15 days prior to Fontan surgical palliation, unless complete and full cardiac recovery is documented by site investigator.
• severe pulmonary hypertension (reported in the medical record as >70% systemic pressure)
• Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe complications or very poor outcome, not directly related tot he stem cell product or its injection procedure, during or after Fontan surgical palliation.
• Individuals with severe heart failure that requires heart transplantation
• Individuals with refractory or worsening arrhythmia
• Individuals with an automated implantable cardioverter defibrillator (AICD) or pacemaker
• Patient with prior surgical complications during the Fontan surgical palliation that resulted in or could be reasonably expected to significantly decrease cardiac function

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mayo Clinic

OTHER

Sponsor Role: collaborator

University of Oklahoma

OTHER

Sponsor Role: collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role: collaborator

Children's Hospital Los Angeles

OTHER

Sponsor Role: collaborator

Children's Hospitals and Clinics of Minnesota

OTHER

Sponsor Role: collaborator

Children's Hospital Colorado

OTHER

Sponsor Role: collaborator

Ochsner Health System

OTHER

Sponsor Role: collaborator

Children's of Alabama

OTHER

Sponsor Role: collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: collaborator

The Hospital for Sick Children

OTHER

Sponsor Role: collaborator

Timothy J Nelson, MD, PhD

OTHER

Sponsor Role: lead

Responsible Party

Timothy J Nelson, MD, PhD

Program Director

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Joseph Dearani

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Harold M Burkhart

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Oklahoma University Medical Center

Joseph w Rossano

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

David M Overman

Role: PRINCIPAL_INVESTIGATOR

Children's Minnesota

John D Cleveland, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Los Angeles

James Jaggers, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Colorado

Benjamin Peeler, MD

Role: PRINCIPAL_INVESTIGATOR

Ochsner Health System

Waldemer Carlo, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Alabama at Birmingham

James Tweddell, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Jason Maynes, MD

Role: PRINCIPAL_INVESTIGATOR

The Hospital forSick Children

Locations

University of Alabama Medical Center

Birmingham, Alabama, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Mayo Clinic

Rochester, Minnesota, United States

University of Oklahoma Medical Center

Oklahoma City, Oklahoma, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

21-000088

Identifier Type: -

Identifier Source: org_study_id