The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

Joshua M Hare25 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hypoplastic Left Heart Syndrome

Interventions

c-kit+ cellsBIOLOGICAL

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.

Eligibility

Sex: ALLMin age: 1 DayMax age: 21 Days

Medical Language ↔ Plain English

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).

Locations (4)

Emory University Children's Healthcare of Atlanta - Egleston Campus

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

University of Maryland - Division of Cardiac Surgery

Baltimore, Maryland, United States

Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

Outcomes

Primary Outcomes

Number of Incidence of Treatment Related Major Adverse Cardiac Events

Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.

Time frame: 30 days

Number of C-kit+ Products

Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects

Time frame: Day 1

Number of Participants Completing Magnetic Resonance Imaging (MRI)

Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.

Time frame: Baseline, 6 Months, 12 months

Change in Right Ventricular Function (RVEF)

Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.

Time frame: Baseline, 6 Months, 12 months

Change in Right Ventricular End-diastolic Volume (RVEDV)

Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.

Time frame: Baseline, 6 months, 12 months

Change in Right Ventricular End-systolic Volume (RVESV)

Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.

Data from ClinicalTrials.gov

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Secondary Outcomes

Number of Incidence of Serious Adverse Events

Incidence of the following after the BDCPA / GLENN Procedure including: All-cause mortality; Cardiovascular mortality; Need for transplantation; Hospitalization for heart failure; Cardiovascular morbidity, including stroke or heart failure or sustained/symptomatic arrhythmias.

Time frame: Up to 12 months

Change in Somatic Growth Velocity - Length (cm)

Changes in somatic growth velocity will be evaluated by length (cm) over 12 months post study product injection.

Time frame: Baseline, 6 months, 12 months

Change in Somatic Growth Velocity - Weight (kg)

Changes in somatic growth velocity will be evaluated by weight (kg) over 12 months post study product injection.

Time frame: Baseline, 6 months, 12 months

Change in Somatic Growth Velocity - Head Circumference (cm)

Changes in somatic growth velocity will be evaluated by head circumference (cm) over 12 months post study product injection.

Time frame: Baseline, 6 months, 12 months

Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health

The Infant Toddler Quality of Life Survey (ITQOL) is a validated 47-item parent-reported questionnaire assessing multiple domains of child health, including overall health, physical abilities, growth and development, discomfort/pain, mood, and behavior. Domain scores are transformed to a 0-100 scale, where 0 reflects the poorest health-related quality of life and 100 reflects the best. Higher scores indicate better quality of life.

Time frame: Baseline, 12 months

Incidence of Mortality or Need for Transplantation

Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported

Time frame: Up to 12 months