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Purpose

The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.

Condition

Eligibility

Eligible Ages
Between 1 Day and 21 Days
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • For inclusion in the study, subjects must meet all of the inclusion criteria: 1. Subjects with hypoplastic left heart syndrome (HLHS) (all types) requiring Stage I Norwood operation.

Exclusion Criteria

  • Candidates will be excluded from the study if any of the following conditions are met: 1. Subjects undergoing the Stage I Norwood operation who do not have HLHS. 2. Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days). 3. Parent or guardian unwilling or unable to comply with necessary follow-up(s). 4. Mother is serum positive for HIV 1/2, hepatitis B surface antigen or viremic hepatitis C and Treponema pallidum. 5. Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
The first 10 participants will be enrolled in Group A to assess safety and feasibility. An additional 22 participants will be enrolled in Group B and will be randomized to either the Treatment or Control Group.
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Open label C-kit+ cells Group A 		Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
  • Biological: c-kit+ cells
    The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
    Other names:
    • Autologous c-kit-positive cells (c-kit+ cells)
Active Comparator
C-kit+ cells Group B 		Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
  • Biological: c-kit+ cells
    The autologous c-kit+ cells will be harvested from participant's right atrial tissue obtained from participant's SOC Norwood Operation. The harvested c-kit+ cells containing up to a total of 12,500 cells/kg will be delivered through 6-10 intramyocardial injections of approximately 100uL per injection for a total volume of approximately 0.6 mL.
    Other names:
    • Autologous c-kit-positive cells (c-kit+ cells)
No Intervention
No Intervention Group 		Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.

More Details

Status
Completed
Sponsor
Joshua M Hare

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.