The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

Phase 1

Completed

• Conditions: Hypoplastic Left Heart Syndrome
• Interventions: Biological: c-kit+ cells

• Registration Number: NCT03406884
• Lead Sponsor: Joshua M Hare

Brief 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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2017-12-20
• Study First Submitted QC: 2018-01-19
• Study First Posted: 2018-01-23
• Study Start: 2019-10-16
• Primary Completion: 2024-07-01
• Study Completion: 2024-07-01
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-22
• Last Update Posted: 2024-11-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

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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).

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
C-kit+ cells Group B	c-kit+ cells	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.
Open label C-kit+ cells Group A	c-kit+ cells	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.

Primary Outcome Measures

Name	Time	Method
Number of c-kit+ products	Day 1	Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Number of participants completing Magnetic Resonance Imaging (MRI)	12 months	Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Change in right ventricular function	Baseline, 12 months	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.
Change in Tricuspid Regurgitation	Baseline, 12 months	Efficacy will be reported as the change tricuspid regurgitation assessed in m/s and will be measured using serial echocardiograms and MRI scan.
Number of Incidence of Treatment Related Major Adverse Cardiac Events	30 days	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.
Change in Right Ventricular End-systolic Volume	Baseline, 12 months	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.
Change in Right Ventricular End-diastolic Volume	Baseline, 12 months	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.

Secondary Outcome Measures

Name	Time	Method
Number of Incidence of Serious Adverse Events	Up to 12 months	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.
Change in somatic growth velocity	Baseline, 12 months	Changes in somatic growth velocity will be evaluated by weight, height, head circumference over 12 months post-SPI.
Change in Infant Toddler Quality of Life Survey (ITQOL)	Baseline, 12 months	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.
Incidence of mortality or need for transplantation	Up to 12 months	Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported

Trial Locations

Locations (3)

Emory University Children's Healthcare of Atlanta - Egleston Campus; 🇺🇸 Atlanta, Georgia, 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