The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

Joshua M Hare

Status and phase: Completed; Phase 1

Conditions: Hypoplastic Left Heart Syndrome

Treatments: Biological: c-kit+ cells

Study type: Interventional

Funder types: Other

Identifiers

NCT03406884

20190743

Details and patient eligibility

About

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.

Enrollment: 25 patients

Sex: All

Ages: 1 to 21 days old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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25 participants in 3 patient groups

Open label C-kit+ cells Group A

Experimental group

Description:

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.

Treatment:

Biological: c-kit+ cells

C-kit+ cells Group B

Active Comparator group

Description:

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.

Treatment:

Biological: c-kit+ cells

No Intervention Group

No Intervention group

Description:

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.