Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome

Completed · Phase 1

Conditions studied: Hypoplastic Left Heart Syndrome

In brief

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.

Key facts

Study ID

NCT01883076

Run by

Timothy J Nelson, MD, PhD

People needed

30

Starts

2013-05-15

Expected to finish

2021-04-28

Last updated by the study team

2022-04-29

Who can join

Age: any, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Children's Hospital of Minnesota — Minneapolis, Minnesota, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Oklahoma University Children's Hospital — Oklahoma City, Oklahoma, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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