The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
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.
- Hypoplastic Left Heart Syndrome
- Eligible Ages
- Between 1 Day and 21 Days
- Eligible Genders
- Accepts Healthy Volunteers
- 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.
- 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).
- Phase 1
- Study Type
- 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
- None (Open Label)
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.||
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.||
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.||
- University of Miami
Study ContactJoshua M Hare, MD