Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome
- Conditions
- Hypoplastic Left Heart Syndrome
- Interventions
- Biological: Autologous (self) mononuclear cells derived from umbilical cord bloodProcedure: Stage II Surgical repair
- Registration Number
- NCT03779711
- Lead Sponsor
- Timothy J Nelson, MD, PhD
- Brief Summary
Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.
- Detailed Description
This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into the myocardium of the right ventricle of the heart at the time of Stage II surgical repair will provide an improvement in cardiac function, reaching growth and developmental milestones, and quality of life, while also providing a reduction in the cumulative days of hospitalization following Stage II surgical repair. Long-term improvement in cardiac function, reaching growth and developmental milestones, reaching Stage III surgical repair pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of life will also be determined.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 95
- Diagnosis of HLHS or HLHS variant with single right ventricular dependent CHD having undergone Stage I surgical repair and undergoing Stage II surgical repair.
- 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)
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History of DMSO reaction (treatment arm only).
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Parent(s) and/or legal guardian(s) unwilling to have their child participate or unwilling to follow the study procedures.
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Severe chronic diseases at the discretion of the treating physician.
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Extensive extra-cardiac syndromic features.
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Known history of cancer.
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Any of the following complications of his/her congenital heart disease:
- 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
- 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 to the stem cell product or its injection procedure, during or after Stage II surgical repair.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Treatment Autologous (self) mononuclear cells derived from umbilical cord blood 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 . Treatment Stage II Surgical repair 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 . Control Stage II Surgical repair 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.
- Primary Outcome Measures
Name Time Method Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC) baseline, 12 months post-Stage II surgery Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Change in Right Ventricular Cardiac Function Measured by Circumferential Strain. baseline, 3 months post-Stage II surgery Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.
Change in Right Ventricular Cardiac Function Measured by Longitudinal Strain. baseline, 3 months post-Stage II surgery Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.
- Secondary Outcome Measures
Name Time Method Change in Right Ventricular Cardiac Function Measured by Apical Fractional Area Change (FAC) baseline, Stage III surgery pre-op Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
Cumulative Days of Hospitalization 3-months post Stage II surgery Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.
Change in Weight baseline, every 6 months post-Stage II surgery Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.
Change in Heart Rate baseline, 3-months post Stage II surgery Change in heart rate between baseline and 3-months post Stage II surgery. Heart rate as measured at home by subject's caregiver as standard of care per subject's provider. Caregivers will be instructed by the site staff on the best way to perform.
Change in Oxygen Saturation baseline, 3-months post Stage II surgery Change in oxygen saturation between baseline and 3-months post Stage II surgery. Oxygen saturation as measured at home by subject's caregiver using the pulse oximetry device provided as standard of care by subject's provider. Caregivers will be instructed by the site to use the same pulse oximetry device for every home measurement of oxygen saturation.
Change in Arrhythmia and Heart Failure Medication 3-months post-surgery Changes in arrhythmia and heart failure medications will be recorded by caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.
Stage III Surgical Repair Stage III surgery pre-op, approx. 4 years The number of research subjects eligible for Stage III surgical repair
Total Time Until Listed for Cardiac Transplantation up to Stage III surgery pre-op, approx. 4 years The number of days between Stage II surgery and listed on cardiac transplantation list.
Total Time Until Death approx 4 years The number of days between Stage II surgery and death occurrence
Trial Locations
- Locations (8)
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
Children's of Alabama🇺🇸Birmingham, Alabama, United States