The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

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 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

  • Hypoplastic Left Heart Syndrome

Eligibility

Eligible Ages
Between 1 Day and 21 Days
Eligible Genders
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 (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 BsAg 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