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

NCT04907526 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 21-000088
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 6

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.

Conditions

Congenital Heart Disease, SRV Dependent

Keywords

Hypoplastic Left Heart Syndrome; HLHS; Congenital Heart Disease; Umbilical Cord Blood; UCB; Cord Blood; Stem Cells; Regenerative therapy; Stage III Fontan; Fontan surgery

Outcome Measures

Primary Outcomes (1)

• Short-term safety

  Measure of new or worsening adverse events

  Within 3 months post Fontan surgery

Secondary Outcomes (8)

• Long term safety

  Within 2 years post Fontan surgery

• Right ventricular function

  Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery

• High sensitivity Troponin T

  3 hours post enrollment, 6 hours post enrollment, hospital discharge (up to 30 days post Fontan surgery)

• NT-pro-BNP

  Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery

• Panel Reactive Antibody

  Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery

Study Arms (2)

Treatment Arm

EXPERIMENTAL

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.

Biological: Autologous mononuclear cells

Control Arm

NO INTERVENTION

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.

Interventions

Autologous mononuclear cells BIOLOGICAL

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

Treatment Arm

Eligibility Criteria

• Age: 2 Years - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Timothy J Nelson, MD, PhD: lead
• Mayo Clinic: collaborator
• University of Oklahoma: collaborator
• Children's Hospital of Philadelphia: collaborator
• Children's Hospital Los Angeles: collaborator
• Children's Hospitals and Clinics of Minnesota: collaborator
• Children's Hospital Colorado: collaborator
• Ochsner Health System: collaborator
• Children's of Alabama: collaborator
• Children's Hospital Medical Center, Cincinnati: collaborator
• The Hospital for Sick Children: collaborator

Study Sites (6)

University of Alabama Medical Center

Birmingham, Alabama, 35233, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Oklahoma Medical Center

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Heart Defects, Congenital; Hypoplastic Left Heart Syndrome

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Joseph Dearani

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

• Harold M Burkhart

  Children's Hospital Oklahoma University Medical Center

  PRINCIPAL INVESTIGATOR

• Joseph w Rossano

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

• David M Overman

  Children's Minnesota

  PRINCIPAL INVESTIGATOR

• John D Cleveland, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

• James Jaggers, MD

  Children's Hospital Colorado

  PRINCIPAL INVESTIGATOR

• Benjamin Peeler, MD

  Ochsner Health System

  PRINCIPAL INVESTIGATOR

• Waldemer Carlo, M.D.

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

• James Tweddell, M.D.

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

• Jason Maynes, MD

  The Hospital forSick Children

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Program Director

Study Record Dates

• First Submitted: May 12, 2021
• First Posted: May 28, 2021
• Study Start: June 1, 2021
• Primary Completion: May 24, 2023
• Study Completion: August 30, 2025
• Last Updated: June 27, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)