The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

Hypoplastic Left Heart Syndrome Clinical Trial

— CHILD  

Official title:

Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03406884
• Other study ID #: 20190743
• Status: Recruiting
• Phase: Phase 1
• Start date: October 16, 2019
• Est. completion date: July 2025

Study information

• Verified date: August 2023
• Source: University of Miami
• Contact: Joshua M Hare, MD
• Phone: 305-243-5779
• Email: JHare[@]med.miami.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 32
• Est. completion date: July 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 21 Days

Eligibility

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).

Related Conditions & MeSH terms

• Hypoplastic Left Heart Syndrome
• Syndrome

Intervention

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.

Locations

Country	Name	City	State
United States	Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital	Ann Arbor	Michigan
United States	Emory University Children's Healthcare of Atlanta - Egleston Campus	Atlanta	Georgia
United States	University of Maryland - Division of Cardiac Surgery	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
University of Miami

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	30 days
Primary	Number of c-kit+ products	Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects	Day 1
Primary	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.	12 months
Primary	Change in right ventricular function	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.	Baseline, 12 months
Primary	Change in Right Ventricular End-diastolic Volume	Efficacy will be reported as the change in right ventricular end-diastolic assessed in mL and will be measured using serial echocardiograms and MRI scan.	Baseline, 12 months
Primary	Change in Right Ventricular End-systolic Volume	Efficacy will be reported as the change in right ventricular end-systolic volume assessed in mm and will be measured using serial echocardiograms and MRI scan.	Baseline, 12 months
Primary	Change in Tricuspid Regurgitation	Efficacy will be reported as the change tricuspid regurgitation assessed in m/s and will be measured using serial echocardiograms and MRI scan.	Baseline, 12 months
Secondary	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.	Up to 12 months
Secondary	Change in somatic growth velocity	Changes in somatic growth velocity will be evaluated by weight, height, head circumference over 12 months post-SPI.	Baseline, 12 months
Secondary	Change in Infant Toddler Quality of Life Survey (ITQOL)	Change in Infant Toddler Quality of Life Survey (ITQOL) is measured on a 9 scale with 47 items measuring child's overall health, physical abilities, overall growth and development, discomfort/pain, moods, and overall behavior. The total score ranges from 0 to 100 with the higher score indicating better quality of life.	Baseline, 12 months
Secondary	Incidence of mortality or need for transplantation	Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported	Up to 12 months

External Links

•   Interdisciplinary stem cell institute at the University of Miami website