Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
- Conditions
- Hypoplastic Left Heart Syndrome
- Interventions
- Biological: autologous cell-based delivery
- Registration Number
- NCT01883076
- Lead Sponsor
- Timothy J Nelson, MD, PhD
- Brief Summary
This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of Hypoplastic Left Heart Syndrome (HLHS) children undergoing a scheduled Glenn surgical procedure.
The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.
- Detailed Description
This study is a Phase I trial to determine the safety of autologous mononuclear cells (MNC) derived from umbilical cord blood for intramyocardial delivery into the right ventricle during a planned and non-emergent Stage II surgical palliation in subjects with HLHS. This is the first critical step towards applying autologous MNC therapy as an add-on regenerative intervention for congenital heart disease management. The choice of HLHS as the target disease for regenerative therapies in congenital heart disease management is multi-factorial and includes the following considerations: 1) Severity of of this incurable disease, 2) palliative nature and burden of long-term outcomes with a single right ventricular system, 3) three stages of planned surgical procedures that provide time points to adjunctively intervene, and 4) prenatal diagnosis enabling planned collection of UCB. An emerging goal for cardiac regeneration includes the application of cell-based technology to congenital heart disease, which is a favorable substrate due to the lack of fibrotic scaring, and the presence of a microenvironment that is expected to support ongoing cardiac proliferation and growth for functional remuscularization. This Phase I safety study will determine the feasibility of collection, processing, and delivery of autologous cells as used in adult cardiac regenerative protocols in the setting of HLHS surgical management.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 30
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description autologous cell-based delivery 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.
- Primary Outcome Measures
Name Time Method Incidence of new and worsening adverse cardiac events Within 2 years following cell therapy treatment The adverse cardiac events would include sustained/symptomatic ventricular arrhythmias, heart failure, myocardial infarction, cardiac infections, and unexpected cardiovascular surgery.
Percentage of subjects enrolled who undergo cell therapy treatment Up to 2 years Incidence of all-cause mortality Within 2 years following cell therapy treatment Percentage of subjects whose cells meet all cell release criteria Up to 2 years
- Secondary Outcome Measures
Name Time Method Change in right ventricular ejection fraction at 3 months according to cardiac imaging with echocardiography baseline, 3 months Change in right ventricle tricuspid annular plane systolic excursion (TAPSE) at one month according to cardiac imaging with echocardiography baseline, 1 month Change in right ventricle TAPSE at 6 months according to cardiac imaging with echocardiography baseline, 6 months Change in right ventricle fractional area change at 6 months according to cardiac imaging with echocardiography baseline, 6 months Change in right ventricular ejection fraction at one month according to cardiac imaging with echocardiography baseline, 1 month Change in right ventricular ejection fraction at 6 months according to cardiac imaging with echocardiography baseline, 6 months Change in right ventricle TAPSE at 3 months according to cardiac imaging with echocardiography baseline, 3 months Change in right ventricle fractional area change at 3 months according to cardiac imaging with echocardiography baseline, 3 months Change in right ventricle fractional area change at one month according to cardiac imaging with echocardiography baseline, 1 month
Trial Locations
- Locations (6)
Children's Hospital of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Oklahoma University Children's Hospital
🇺🇸Oklahoma City, Oklahoma, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States