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The CHILD Trial: Hypoplastic Left Heart Syndrome Study. (CHILD)

Primary Purpose

Hypoplastic Left Heart Syndrome

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
c-kit+ cells
Sponsored by
University of Miami
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypoplastic Left Heart Syndrome focused on measuring stem cells, pediatric

Eligibility Criteria

1 Day - 21 Days (Child)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Emory University Children's Healthcare of Atlanta - Egleston CampusRecruiting
  • University of Maryland - Division of Cardiac SurgeryRecruiting
  • Michigan Medicine Congenital Heart Center/C.S. Mott Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Active Comparator

No Intervention

Arm Label

Open label C-kit+ cells Group A

C-kit+ cells Group B

No Intervention Group

Arm 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.

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.

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.

Outcomes

Primary Outcome Measures

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.
Number of c-kit+ products
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)
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
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 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.
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.
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.

Secondary Outcome Measures

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.
Change in somatic growth velocity
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)
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
Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported

Full Information

First Posted
December 20, 2017
Last Updated
August 11, 2023
Sponsor
University of Miami
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1. Study Identification

Unique Protocol Identification Number
NCT03406884
Brief Title
The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
Acronym
CHILD
Official Title
Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 16, 2019 (Actual)
Primary Completion Date
July 2025 (Anticipated)
Study Completion Date
July 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Miami

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypoplastic Left Heart Syndrome
Keywords
stem cells, pediatric

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
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.
Masking
None (Open Label)
Allocation
Randomized
Enrollment
32 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Open label C-kit+ cells Group A
Arm Type
Experimental
Arm 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.
Arm Title
C-kit+ cells Group B
Arm Type
Active Comparator
Arm 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.
Arm Title
No Intervention Group
Arm Type
No Intervention
Arm 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.
Intervention Type
Biological
Intervention Name(s)
c-kit+ cells
Other Intervention Name(s)
Autologous c-kit-positive cells (c-kit+ cells)
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Incidence of Treatment Related Major Adverse Cardiac Events
Description
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
Title
Number of c-kit+ products
Description
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Time Frame
Day 1
Title
Number of participants completing Magnetic Resonance Imaging (MRI)
Description
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Time Frame
12 months
Title
Change in right ventricular function
Description
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, 12 months
Title
Change in Right Ventricular End-diastolic Volume
Description
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.
Time Frame
Baseline, 12 months
Title
Change in Right Ventricular End-systolic Volume
Description
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.
Time Frame
Baseline, 12 months
Title
Change in Tricuspid Regurgitation
Description
Efficacy will be reported as the change tricuspid regurgitation assessed in m/s and will be measured using serial echocardiograms and MRI scan.
Time Frame
Baseline, 12 months
Secondary Outcome Measure Information:
Title
Number of Incidence of Serious Adverse Events
Description
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
Title
Change in somatic growth velocity
Description
Changes in somatic growth velocity will be evaluated by weight, height, head circumference over 12 months post-SPI.
Time Frame
Baseline, 12 months
Title
Change in Infant Toddler Quality of Life Survey (ITQOL)
Description
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.
Time Frame
Baseline, 12 months
Title
Incidence of mortality or need for transplantation
Description
Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported
Time Frame
Up to 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Day
Maximum Age & Unit of Time
21 Days
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: For inclusion in the study, subjects must meet all of the inclusion criteria: 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: Subjects undergoing the Stage I Norwood operation who do not have HLHS. Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days). Parent or guardian unwilling or unable to comply with necessary follow-up(s). Mother is serum positive for HIV 1/2, hepatitis BsAg or viremic hepatitis C and Treponema pallidum. Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Joshua M Hare, MD
Phone
305-243-5779
Email
JHare@med.miami.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Yvenie Desire, BA
Phone
305-243-7273
Email
YDesire@miami.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William Mahle, MD
Organizational Affiliation
Emory University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Kristopher Deatrick, MD
Organizational Affiliation
University of Maryland
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Richard Ohye, MD
Organizational Affiliation
Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Emory University Children's Healthcare of Atlanta - Egleston Campus
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristen Herzegh, MPH
Phone
404-712-7596
Email
kcoshau@emory.edu
First Name & Middle Initial & Last Name & Degree
Michael Davis, PhD
Facility Name
University of Maryland - Division of Cardiac Surgery
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristopher Deatrick, MD
Phone
410-328-5842
Email
KDeatrick@som.umaryland.edu
First Name & Middle Initial & Last Name & Degree
Manal Al-Suqi, MSTC
Phone
410-328-9409
Email
MaAl-Suqi@som.umaryland.edu
First Name & Middle Initial & Last Name & Degree
Kristopher Deatrick
Facility Name
Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tammy Doman, RN
Phone
734-763-6109
Email
tpaterso@med.umich.edu
First Name & Middle Initial & Last Name & Degree
Richard Ohye, MD

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
35394149
Citation
Kaushal S, Hare JM, Shah AM, Pietris NP, Bettencourt JL, Piller LB, Khan A, Snyder A, Boyd RM, Abdullah M, Mishra R, Sharma S, Slesnick TC, Si MS, Chai PJ, Davis BR, Lai D, Davis ME, Mahle WT. Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study). Pediatr Cardiol. 2022 Oct;43(7):1481-1493. doi: 10.1007/s00246-022-02872-6. Epub 2022 Apr 8.
Results Reference
derived
PubMed Identifier
34217109
Citation
Ali MK, Ichimura K, Spiekerkoetter E. Promising therapeutic approaches in pulmonary arterial hypertension. Curr Opin Pharmacol. 2021 Aug;59:127-139. doi: 10.1016/j.coph.2021.05.003. Epub 2021 Jun 30.
Results Reference
derived
Links:
URL
http://ISCI.MED.MIAMI.EDU
Description
Interdisciplinary stem cell institute at the University of Miami website

Learn more about this trial

The CHILD Trial: Hypoplastic Left Heart Syndrome Study.

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