Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
Hypoplastic Left Heart Syndrome
About this trial
This is an interventional treatment trial for Hypoplastic Left Heart Syndrome focused on measuring Hypoplastic Left Heart Syndrome, HLHS, Congenital Heart Disease, Umbilical cord blood, UCB, Cord blood, Stem cells, Regenerative therapy, Stage II Glenn, Glenn Surgery
Eligibility Criteria
Inclusion Criteria
Individuals with autologous cord blood product that met all cell release criteria (listed on the certificate of analysis from Mayo Clinic Human Cell Therapy Lab) as follows:
- No aerobic or anaerobic bacterial growth after 14 days
- Greater than 70% cell viability pre-freeze
- Total Nucleated Cells (TNC) concentration of 30-42 x 106 cells/mL (pre-freeze)
- Minimum of one (1) vial of cells
- Mononuclear cell percentage of greater than 50%
- Endotoxin result of less than 16 Endotoxin Units (EU)/mL.
- Mother's serology test results are negative for HIV, Hepatitis B, and Hepatitis C.
- Individuals with HLHS having undergone Stage I surgical palliation and undergoing planned Stage II palliative Glenn surgery.
- Ages up to 18 months are eligible if written informed consent can be obtained from both parents (unless one parent is not reasonably available) and/or legal guardians.
Exclusion Criteria
- Child who's UCB does not meet the specified cell release criteria in Inclusion Criterion #1.
- History of dimethyl sulfoxide (DMSO) reaction for either the child or mother.
- Parent(s)/child unwilling to participate.
- Child with severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer.
- Child not completing all pre-procedure work-up within 10 days of the Stage II Glenn surgery as listed in section 6 of this protocol AND lack of pre-procedure work-up documented as a safety concern by a site investigator.
- Child who's cells have been compromised after meeting cell release criteria (as defined in Inclusion Criterion #1).
Child with the following complications of their congenital heart disease:
- Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage II surgical repair
- 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 during or after Stage II surgical repair.
Sites / Locations
- Children's Hospital Los Angeles
- Children's Hospital Colorado
- Children's Hospital of Minnesota
- Mayo Clinic
- Oklahoma University Children's Hospital
- Children's Hospital of Philadelphia
Arms of the Study
Arm 1
Experimental
autologous cell-based delivery
autologous cell-based delivery a target dose of 3 million cells / kg of body weight will be delivered into the right heart muscle at the time of surgery. Cells are derived from autologous (self) umbilical cord blood.