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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03406884
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : February 21, 2020
Sponsor:
Information provided by (Responsible Party):
Joshua M Hare, University of Miami

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 Bidirectional Cavopulmonary Anastomosis (BDCPA/GLENN) Procedure and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, hospitalizations for heart failure, changes in health status from baseline, the need for transplantation, or mortality.

Condition or disease Intervention/treatment Phase
Hypoplastic Left Heart Syndrome Biological: c-kit+ cells Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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)
Primary Purpose: Treatment
Official Title: Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
Actual Study Start Date : October 16, 2019
Estimated Primary Completion Date : July 2025
Estimated Study Completion Date : July 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Group A Safety and Feasibility Open Label Group
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 BDCPA/GLENN operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
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.
Other Name: Autologous c-kit-positive cells (c-kit+ cells)

Active Comparator: Group B Treatment Group.
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their BDCPA/GLENN operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
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.
Other Name: Autologous c-kit-positive cells (c-kit+ cells)

No Intervention: Group B Control Group
Participants randomized to Group B Control Group will receive only their standard of care (SOC) BDCPA/GLENN procedure without the injection of harvested c-kit+ cells.



Primary Outcome Measures :
  1. 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.

  2. 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

  3. 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.

  4. 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.

  5. Change in Right Ventricular Volume [ Time Frame: Baseline, 12 months ]
    Efficacy will be reported as the change in right ventricular end-diastolic and end-systolic volume assessed in mL and will be measured using serial echocardiograms and MRI scan.

  6. Change in Right Ventricular End-Systolic Diameter [ Time Frame: Baseline, 12 months ]
    Efficacy will be reported as the change in right ventricular end-systolic diameter assessed in mm and will be measured using serial echocardiograms and MRI scan.

  7. 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.


Secondary Outcome Measures :
  1. Incidence of Mortality or Transplantation [ Time Frame: Baseline, 12 months ]
    The number of incidence of mortality or need for transplantation after the BDCPA/GLENN procedure will be reported.

  2. Number of Incidence of Serious Adverse Events [ Time Frame: Baseline, 12 months ]
    Number of incidence of the following serious adverse events after the BDCPA/GLENN procedure will be reported: all cause mortality, cardiovascular mortality, need for transplantation, hospitalization for heart failure and cardiovascular morbidity including stroke, heart failure or sustained/symptomatic arrhythmias.

  3. Change in somatic growth velocity [ Time Frame: Baseline, 12 months ]
    Changes in somatic growth velocity will be evaluated.



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Ages Eligible for Study:   up to 21 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 prior to surgery.
    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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03406884


Contacts
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Contact: Joshua M Hare, MD 305-243-5779 JHare@med.miami.edu
Contact: Yvenie Desire, BA 305-243-7273 YDesire@miami.edu

Locations
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United States, Georgia
Emory University Children's Healthcare of Atlanta - Egleston Campus Recruiting
Atlanta, Georgia, United States, 30322
Contact: Kristen Herzegh, MPH    404-712-7596    kcoshau@emory.edu   
Sub-Investigator: Michael Davis, PhD         
United States, Maryland
University of Maryland - Division of Cardiac Surgery Recruiting
Baltimore, Maryland, United States, 21201
Contact: Sunjay Kaushal, MD    410-328-5842      
Contact: Manal Al-Suqi, MSTC    410-328-9409    MaAl-Suqi@som.umaryland.edu   
Sponsors and Collaborators
Joshua M Hare
Investigators
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Principal Investigator: Michael Davis, PhD Emory University
Principal Investigator: Sunjay Kaushal, MD University of Maryland, College Park
Principal Investigator: Joshua M Hare, MD University of Miami

Additional Information:
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Responsible Party: Joshua M Hare, Professor, University of Miami
ClinicalTrials.gov Identifier: NCT03406884    
Other Study ID Numbers: 20190743
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: February 21, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Joshua M Hare, University of Miami:
stem cells
pediatric
Additional relevant MeSH terms:
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Hypoplastic Left Heart Syndrome
Syndrome
Disease
Pathologic Processes
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities