- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03406884
The CHILD Trial: Hypoplastic Left Heart Syndrome Study. (CHILD)
August 11, 2023 updated by: University of Miami
Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
32
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Yvenie Desire, BA
- Phone Number: 305-243-7273
- Email: YDesire@miami.edu
Study Contact Backup
- Name: Joshua M Hare, MD
- Phone Number: 305-243-5779
- Email: JHare@med.miami.edu
Study Locations
-
-
Georgia
-
Atlanta, Georgia, United States, 30322
- Recruiting
- Emory University Children's Healthcare of Atlanta - Egleston Campus
-
Contact:
- Kristen Herzegh, MPH
- Phone Number: 404-712-7596
- Email: kcoshau@emory.edu
-
Sub-Investigator:
- Michael Davis, PhD
-
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Maryland
-
Baltimore, Maryland, United States, 21201
- Recruiting
- University of Maryland - Division of Cardiac Surgery
-
Contact:
- Manal Al-Suqi, MSTC
- Phone Number: 410-328-9409
- Email: MaAl-Suqi@som.umaryland.edu
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Contact:
- Kristopher Deatrick, MD
- Phone Number: 410-328-5842
- Email: KDeatrick@som.umaryland.edu
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Principal Investigator:
- Kristopher Deatrick
-
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Michigan
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Ann Arbor, Michigan, United States, 48109
- Recruiting
- Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
-
Contact:
- Tammy Doman, RN
- Phone Number: 734-763-6109
- Email: tpaterso@med.umich.edu
-
Principal Investigator:
- Richard Ohye, MD
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 day to 3 weeks (Child)
Accepts Healthy Volunteers
No
Description
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).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Open label C-kit+ cells Group A
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.
|
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.
Other Names:
|
Active Comparator: C-kit+ cells Group B
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.
|
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.
Other Names:
|
No Intervention: No Intervention Group
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Incidence of Treatment Related Major Adverse Cardiac Events
Time Frame: 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.
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30 days
|
Number of c-kit+ products
Time Frame: Day 1
|
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
|
Day 1
|
Number of participants completing Magnetic Resonance Imaging (MRI)
Time Frame: 12 months
|
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
|
12 months
|
Change in right ventricular function
Time Frame: 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.
|
Baseline, 12 months
|
Change in Right Ventricular End-diastolic Volume
Time Frame: 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.
|
Baseline, 12 months
|
Change in Right Ventricular End-systolic Volume
Time Frame: 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.
|
Baseline, 12 months
|
Change in Tricuspid Regurgitation
Time Frame: 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.
|
Baseline, 12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Incidence of Serious Adverse Events
Time Frame: 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.
|
Up to 12 months
|
Change in somatic growth velocity
Time Frame: Baseline, 12 months
|
Changes in somatic growth velocity will be evaluated by weight, height, head circumference over 12 months post-SPI.
|
Baseline, 12 months
|
Change in Infant Toddler Quality of Life Survey (ITQOL)
Time Frame: 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.
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Baseline, 12 months
|
Incidence of mortality or need for transplantation
Time Frame: Up to 12 months
|
Incidence of mortality or need for transplantation after the Stage II BDCPA operation will be reported
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Up to 12 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Kristopher Deatrick, MD, University of Maryland
- Principal Investigator: Richard Ohye, MD, Michigan Medicine Congenital Heart Center/C.S. Mott Children's Hospital
- Principal Investigator: William Mahle, MD, Emory University
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 16, 2019
Primary Completion (Estimated)
July 1, 2025
Study Completion (Estimated)
July 1, 2025
Study Registration Dates
First Submitted
December 20, 2017
First Submitted That Met QC Criteria
January 19, 2018
First Posted (Actual)
January 23, 2018
Study Record Updates
Last Update Posted (Actual)
August 14, 2023
Last Update Submitted That Met QC Criteria
August 11, 2023
Last Verified
August 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 20190743
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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