Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syn… (NCT01883076) | Clinical Trial Compass
CompletedPhase 1
Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
United States30 participantsStarted 2013-05-15
Plain-language 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.
Who can participate
Age range
18 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of all-cause mortality
Timeframe: Within 2 years following cell therapy treatment
2
Incidence of new and worsening adverse cardiac events
Timeframe: Within 2 years following cell therapy treatment
3
Percentage of subjects whose cells meet all cell release criteria
Timeframe: Up to 2 years
4
Percentage of subjects enrolled who undergo cell therapy treatment
. Mother's serology test results are negative for HIV, Hepatitis B, and Hepatitis C.
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