Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome
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ClinicalTrials.gov Identifier: NCT01883076 |
Recruitment Status :
Active, not recruiting
First Posted : June 21, 2013
Last Update Posted : October 5, 2020
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Tracking Information | ||||||||||||||||||||||
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First Submitted Date ICMJE | June 11, 2013 | |||||||||||||||||||||
First Posted Date ICMJE | June 21, 2013 | |||||||||||||||||||||
Last Update Posted Date | October 5, 2020 | |||||||||||||||||||||
Actual Study Start Date ICMJE | May 15, 2013 | |||||||||||||||||||||
Estimated Primary Completion Date | November 2020 (Final data collection date for primary outcome measure) | |||||||||||||||||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
Change in right ventricular ejection fraction according to cardiac imaging with echocardiography [ Time Frame: 6 months follow-up post Glenn procedure ] The secondary analysis is focused on the potential benefit of intramyocardial delivery of the manufactured cell-based product in pediatric hearts at the time of planned surgical palliation for HLHS. The change from baseline to 1, 3, and 6-month follow-up in right ventricular ejection fraction is the primary variable along with secondary variables of change in right ventricle TAPSE and fractional area change.
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Current Other Pre-specified Outcome Measures | Not Provided | |||||||||||||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | |||||||||||||||||||||
Descriptive Information | ||||||||||||||||||||||
Brief Title ICMJE | Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome | |||||||||||||||||||||
Official Title ICMJE | Phase I Safety Study of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Stage II Palliation of Hypoplastic Left Heart Syndrome | |||||||||||||||||||||
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. |
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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. | |||||||||||||||||||||
Study Type ICMJE | Interventional | |||||||||||||||||||||
Study Phase ICMJE | Phase 1 | |||||||||||||||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Hypoplastic Left Heart Syndrome | |||||||||||||||||||||
Intervention ICMJE | Biological: autologous cell-based delivery
autologous cells (derived from "self")
Other Name: umbilical cord blood derived mononuclear cells
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Study Arms ICMJE | 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.
Intervention: Biological: autologous cell-based delivery
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Publications * | Not Provided | |||||||||||||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||||||||||||||||||||
Recruitment Status ICMJE | Active, not recruiting | |||||||||||||||||||||
Estimated Enrollment ICMJE |
30 | |||||||||||||||||||||
Original Estimated Enrollment ICMJE |
10 | |||||||||||||||||||||
Estimated Study Completion Date ICMJE | May 2021 | |||||||||||||||||||||
Estimated Primary Completion Date | November 2020 (Final data collection date for primary outcome measure) | |||||||||||||||||||||
Eligibility Criteria ICMJE | Inclusion Criteria
Exclusion Criteria
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Sex/Gender ICMJE |
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Ages ICMJE | up to 18 Months (Child) | |||||||||||||||||||||
Accepts Healthy Volunteers ICMJE | No | |||||||||||||||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||||||||||||||||||||
Listed Location Countries ICMJE | United States | |||||||||||||||||||||
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Administrative Information | ||||||||||||||||||||||
NCT Number ICMJE | NCT01883076 | |||||||||||||||||||||
Other Study ID Numbers ICMJE | 12-008521 | |||||||||||||||||||||
Has Data Monitoring Committee | Yes | |||||||||||||||||||||
U.S. FDA-regulated Product | Not Provided | |||||||||||||||||||||
IPD Sharing Statement ICMJE |
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Responsible Party | Timothy J Nelson, MD, PhD, ReGen Theranostics, Inc. | |||||||||||||||||||||
Study Sponsor ICMJE | Timothy J Nelson, MD, PhD | |||||||||||||||||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | ReGen Theranostics, Inc. | |||||||||||||||||||||
Verification Date | October 2020 | |||||||||||||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |