Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome
NCT ID: NCT03779711
Last Updated: 2025-07-08
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE2
95 participants
INTERVENTIONAL
2019-06-06
2026-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Treatment
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 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .
Autologous (self) mononuclear cells derived from umbilical cord blood
The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.
Stage II Surgical repair
This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.
Control
Clinical data will be collected before, during and after the Stage II surgical standard of care procedure. Requires additional imaging studies at 3 months that are not standard of care in addition to some blood tests that would be beyond standard of care. Information will be collected to document the clinical outcomes and will be compared with the information from the group receiving the investigational treatment used in this study.
Stage II Surgical repair
This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.
Interventions
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Autologous (self) mononuclear cells derived from umbilical cord blood
The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.
Stage II Surgical repair
This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.
Eligibility Criteria
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Inclusion Criteria
* Less than 13 months of age at time of Stage II surgical repair
* Previous participation in the UCB collection protocol with autologous UCB-MNC product that is acceptable for use (treatment arm only)
Exclusion Criteria
* Parent(s) and/or legal guardian(s) 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.
* Known history of cancer.
* Any of the following complications of his/her congenital heart disease:
1. Any condition requiring urgent, or unplanned interventional procedure within 15 days prior to Stage II surgical repair, unless complete and full cardiac recovery is documented by site investigator
2. Severe pulmonary hypertension (reported in the medical record as \>70% systemic pressure)
3. 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 to the stem cell product or its injection procedure, during or after Stage II surgical repair.
12 Months
ALL
No
Sponsors
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University of Oklahoma
OTHER
Children's Hospital of Philadelphia
OTHER
Children's Hospital Los Angeles
OTHER
Children's Hospital Colorado
OTHER
Children's Hospitals and Clinics of Minnesota
OTHER
Ochsner Health System
OTHER
Children's of Alabama
OTHER
Children's Hospital Medical Center, Cincinnati
OTHER
Timothy J Nelson, MD, PhD
OTHER
Responsible Party
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Timothy J Nelson, MD, PhD
Program Director
Principal Investigators
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Frank Cetta, MD
Role: PRINCIPAL_INVESTIGATOR
Mayo Clinic
Harold M. Burkhart, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Oklahoma University Medical Center
Joseph W. Rossano, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia
David M. Overman, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Minnesota
Ram Kumar Subramanyan, M.D., Ph.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Los Angeles
James Jaggers, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Colorado
Benjamin Peeler, M.D.
Role: PRINCIPAL_INVESTIGATOR
Ochsner Health System
Waldemar Carlo, M.D.
Role: PRINCIPAL_INVESTIGATOR
University of Alabama at Birmingham
James Tweddell, M.D.
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Locations
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Children's of Alabama
Birmingham, Alabama, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ocshner Medical Center
New Orleans, Louisiana, United States
Children's Hospitals of Minnesota
Minneapolis, Minnesota, United States
Cincinnati Children's Hospital and Medical Center
Cincinnati, Ohio, United States
Oklahoma University Medical Center
Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Countries
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References
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Gallego-Navarro C, Jaggers J, Burkhart HM, Carlo WF, Morales DL, Qureshi MY, Rossano JW, Hagen CE, Seisler DK, Peral SC, Nelson TJ. Autologous umbilical cord blood mononuclear cell therapy for hypoplastic left heart syndrome: a nonrandomized control trial of the efficacy and safety of intramyocardial injections. Stem Cell Res Ther. 2025 May 1;16(1):215. doi: 10.1186/s13287-025-04316-3.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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Mayo Clinic Clinical Trials
Other Identifiers
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18-004753
Identifier Type: -
Identifier Source: org_study_id
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