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| Name | Class |
|---|---|
| Mayo Clinic | OTHER |
| University of Oklahoma | OTHER |
| Children's Hospital of Philadelphia | OTHER |
| Children's Hospital Los Angeles |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous mononuclear cells | Biological | Autologous mononuclear cells delivered into right ventricle at time of Stage III Fontan surgery. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Short-term safety | Measure of new or worsening adverse events | Within 3 months post Fontan surgery |
| Measure | Description | Time Frame |
|---|---|---|
| Long term safety | Measure of new or worsening adverse events | Within 2 years post Fontan surgery |
| Right ventricular function | Change from baseline Transthoracic Echocardiogram right ventricular function as measured by biplane Fractional Area Change (FAC) at discharge and 3 months |
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Inclusion Criteria:
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:
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
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| Name | Affiliation | Role |
|---|---|---|
| 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 |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama Medical Center | Birmingham | Alabama | 35233 | United States | ||
| Children's Hospital Los Angeles |
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| OTHER |
| Children's Hospitals and Clinics of Minnesota | OTHER |
| Children's Hospital Colorado | OTHER |
| Ochsner Health System | OTHER |
| Children's of Alabama | OTHER |
| Children's Hospital Medical Center, Cincinnati | OTHER |
| The Hospital for Sick Children | OTHER |
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| Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery |
| High sensitivity Troponin T | Change from baseline in High sensitivity Troponin T at 3 hours and 6 hours after enrollment and at hospital discharge | 3 hours post enrollment, 6 hours post enrollment, hospital discharge (up to 30 days post Fontan surgery) |
| NT-pro-BNP | Change from baseline in NT-pro-BNP levels compared to baseline, at hospital discharge and at 3 months post-Fontan surgery | Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery |
| Panel Reactive Antibody | Change from baseline in PRA levels at discharge and 3 months post-Fontan surgery compared to baseline. | Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months post Fontan surgery |
| Weight | Change from baseline in weight at 3 and 12 months post-Fontan surgery. | Baseline, 3 months post-Fontan surgery, 12 months post-Fontan surgery |
| Cumulative days hospitalization | Cumulative days of hospitalization per patient at 3 and 12 months post-Fontan surgery discharge | 3 months post discharge, 12 months post discharge |
| PROMIS Parent Proxy Scale v1.0-Global Health 7 | Change from baseline in PROMIS Parent Proxy Scale v1.0-Global Health 7 at discharge, 3, 12, 18, and 24 months post-Fontan surgery | Baseline, hospital discharge (up to 30 days post Fontan surgery), 3 months, 12 months, 18 months, 24 months |
| Jason Maynes, MD |
| The Hospital forSick Children |
| Principal Investigator |
| Los Angeles |
| California |
| 90027 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| University of Oklahoma Medical Center | Oklahoma City | Oklahoma | 73104 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| ID | Term |
|---|---|
| D006330 | Heart Defects, Congenital |
| D018636 | Hypoplastic Left Heart Syndrome |
| ID | Term |
|---|---|
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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