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Trial record 2 of 3 for:    c-kit+

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

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ClinicalTrials.gov Identifier: NCT03406884
Recruitment Status : Not yet recruiting
First Posted : January 23, 2018
Last Update Posted : September 25, 2018
Sponsor:
Collaborator:
University of Maryland
Information provided by (Responsible Party):
Joshua M Hare, University of Miami

Brief Summary:
Male or female neonates, < 28 days (inclusive) diagnosed with hypoplastic left heart syndrome (HLHS) undergoing Stage I surgery. A total of 30 patients will be enrolled for this pilot study in a staged enrollment diagnosed with HLHS.All patients will be followed for 12 months post-treatment.

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

Detailed Description:

All subjects with the diagnosis of hypoplastic left heart syndrome (HLHS) undergo a palliative reconstructive surgery performed in the first two weeks of life, termed the Norwood procedure. The second stage in the palliation of HLHS is a scheduled bidirectional cavopulmonary anastomosis (BDCPA) that occurs between 4 to 6 months of age. During this surgery when the patient is on cardiopulmonary bypass for the BDCPA, the study product will be given from the previously harvested, isolated, and expanded autologous c-kit+ cardiac stem cells into the aorta directly through the cardioplegia needle into the coronary circulation.

In summary, the aim is to overlay a novel cell therapeutic strategy on the two-stage surgical procedures that HLHS patients typically undergo in the first year of life: Stage I Norwood operation in the neonatal period and Stage II BDPCA operation at approximately 4 months of age.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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.
Estimated Study Start Date : October 2018
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 - c-kit+ cells
Group A - c-kit+ / Ten (10) subjects will be treated with c-kit+ cell treatment.
Drug: c-kit+ cells
15 patients will eventually receive intra-coronary injection of the c-kit+ cells in Group A - c-kit+ cells and Group B - c-kit+ cells
Other Name: stem cell

Experimental: Group B - c-kit+ cells
Group B - c-kit+ / Five (5) subjects will be treated with c-kit+ cell treatment.
Drug: c-kit+ cells
15 patients will eventually receive intra-coronary injection of the c-kit+ cells in Group A - c-kit+ cells and Group B - c-kit+ cells
Other Name: stem cell

Placebo Comparator: Group B - Placebo
Group B - Placebo / Ten (10) subjects will be treated with placebo.
Drug: Placebo
15 control patients receive intra-coronary injections with placebo (no cell injection)
Other Name: control drug




Primary Outcome Measures :
  1. Assess c-kit+ cells safety by reviewing treatment-related adverse events.. [ Time Frame: at 12 months ]
    Safety and feasibility of intracoronary delivery of c-kit+ cells in subjects with HLHS after one year of having received investigational product (IP). Assessed by monitoring the number of treatment-related adverse events assessed, but not limited to death, non-fatal events, and hospitalization for worsening symptoms.


Secondary Outcome Measures :
  1. Change in baseline right ventricular function [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular function, measured by serial echocardiograms

  2. Change in baseline right ventricular end-diastolic volume [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-diastolic volume, measured by serial echocardiograms

  3. Change in baseline right ventricular end-systolic volume [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-systolic volume, measured by serial echocardiograms

  4. Change in baseline right ventricular end-systolic diameter [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-systolic diameter, measured by serial echocardiograms

  5. Change in baseline tricuspid regurgitation [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in tricuspid regurgitation, measured by serial echocardiograms

  6. Change in baseline right ventricular function [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular function as measured by MRI Scans

  7. Change in baseline right ventricular end-diastolic volume [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-diastolic volume, as measured by MRI Scans

  8. Change in baseline right ventricular end-systolic volume [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-systolic volume as measured by MRI Scans

  9. Change in baseline right ventricular end-systolic diameter [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in right ventricular end-systolic diameter as measured by MRI Scans

  10. Change in baseline tricuspid regurgitation [ Time Frame: At Baseline, 6 months and 1 year ]
    Change from baseline in tricuspid regurgitation as measured by MRI Scans

  11. Incidence of mortality or need for transplantation after the BDCPA [ Time Frame: Up to the one year follow-up ]
    Incidence of mortality or need for transplantation after the BDCPA up to one year follow-up.

  12. Changes in somatic growth velocity over time [ Time Frame: up to the one year follow-up ]
    Changes in somatic growth velocity over time by assessing weight, height, head circumference from the BDCPA operation out to 12 months.

  13. Assessment of co-morbidity [ Time Frame: up to the one year follow-up ]
    Assessment of co-morbidity by reviewing Cardiovascular mortality, All cause mortality, Cardiovascular morbidity, Re-hospitalizations, and/or the number of participants that need transplantation.



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Ages Eligible for Study:   up to 27 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 < 28 days of age.
  2. Subjects with hypoplastic left heart syndrome (all types) requiring Norwood surgery.

Exclusion Criteria:

Candidates will be excluded from the study if any of the following conditions are met:

  1. Subjects with HLHS and restrictive or intact atrial septum.
  2. Subjects undergoing the Norwood procedure who do not have HLHS.
  3. Subjects with significant coronary artery sinusoids.
  4. Subjects with birth weights of less than 2 kilograms
  5. Subjects requiring mechanical circulatory support prior to surgery
  6. Subjects with underlying evidence of arrhythmia requiring anti-arrhythmia therapy
  7. Subjects who are unwilling or unable to comply with necessary follow-up
  8. Subjects who are unsuitable for inclusion in the study in the opinion of the investigators

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
Contact: Joshua M Hare, MD 305-243-5779 JHare@med.miami.edu
Contact: Marietsy Pujol, MBA 305-243-7273 mpujol@med.miami.edu

Locations
United States, Florida
ISCI / University of Miami Miller School of Medicine Not yet recruiting
Miami, Florida, United States, 33136
Contact: Joshua M Hare, MD    305-243-5579    jhare@med.miami.edu   
Contact: Study Coordinators    305-243-7444    ISCIStudInfo@miami.edu   
United States, Georgia
Emory University Children's Healthcare of Atlanta - Egleston Campus Not yet recruiting
Atlanta, Georgia, United States, 30322
Contact: Kristen Herzegh, MPH    404-712-7596    kcoshau@emory.edu   
Sub-Investigator: Michael Davis, PhD         
Sponsors and Collaborators
Joshua M Hare
University of Maryland
Investigators
Principal Investigator: Michael Davis, PhD Emory University

Additional Information:
Responsible Party: Joshua M Hare, Principal Investigator, University of Miami
ClinicalTrials.gov Identifier: NCT03406884     History of Changes
Other Study ID Numbers: HLHS
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: September 25, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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:
Syndrome
Hypoplastic Left Heart Syndrome
Disease
Pathologic Processes
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities