Phase I Safety Study of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Stage II Palliation of Hypoplastic Left Heart Syndrome

Location:

Rochester, Minn.

Trial status:

Open for Enrollment

Why is this study being done?

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.

Who is eligible to participate?

Inclusion Criteria: - Individuals with autologous cord blood product enrolled, consented and collected under IRB #11-007176, that met all release criteria according to that protocol. - Individuals with HLHS undergoing planned Stage II palliative surgeries. - 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: - Individuals with UCB collected under consent and processed according to IRB #11-007176, not meeting specified release criteria or have not previously collected UCB. - History of DMSO reaction for either the infant or mother. - Individuals with families unwilling to participate. - Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer. - Infants with the following conditions within 15 days prior to the date of the Stage II Glenn surgery: - Cardiogenic shock - Pulmonary hypertension requiring chronic medical therapy (e.g. supplemental oxygen, vasodilator) - Arrhythmia that required medication for control - Any documented infection requiring treatment with IV antibiotics, and/or current infection being treated with antibiotics - Infants with the following complications of their congenital heart disease: - Any cardiac condition requiring urgent, or unplanned procedure 15 days prior to Stage II Glenn surgery - Tricuspid repair and/or aortic arch repair at the time of Stage II Glenn surgery - Length of hospitalization of more than 60 days for Stage I Norwood procedure - Chylothorax requiring dietary modifications - Seizure or neurological injury - Moderate to severe tricuspid regurgitation prior to Stage II Glenn surgery - History of extracorporeal membrane oxygenator (ECMO) support

Last updated:

8/29/2014

NCT ID:

NCT01883076

IRB Number:

12-008521