Clinical Trial: Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.

Brief Summary: This study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.

Detailed Summary:

This is an open label study, intended as a safety and efficacy assessment prior to a full comparator study. In this Phase I study, cells administered will be delivered in 6-10 intramyocardial injections that will be tested in 20 patients and 10 patients will be controls with a total of 30 HLHS patients.

A total of 30 patients with HLHS will be enrolled in a staged enrollment process. In this open-labeled study, a maximum of 20 patients will eventually receive intramyocardial injection of the allogeneic mesenchymal stem cells and 10 control patients with no cell injection. The enrollment of the patients will occur in two stages groups: Group A and Group B. In Group A, 10 consecutive HLHS patients will be initially enrolled in the allogenic MSCs treatment arm to determine feasibility and safety. After 6 months of the last enrolled patient in Group A, all Group A patients will be assessed in order to determine whether it is feasible and safe, including the harvesting, processing, and administering of the allogeneic MSCs. Thereafter, Group B will start enrolling a total of 20 HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio, respectively, in order to have 10 allogeneic MSCs-treated patient and 10 control patients. At the completion of this Phase I clinical study, the total enrolled cohort will be 20 patients treated with allogeneic MSCs and 10 patients in the control arm.


Sponsor: Joshua M Hare

Current Primary Outcome: Monitor major adverse cardiac events [ Time Frame: 1 month after injection ]

Monitoring of events includes death, sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support, aggravation of heart failure, new myocardial infarction, unplanned cardiovascular operation for cardiac tamponade and infection in the first month after injection, and serially afterwards.


Original Primary Outcome: Number of participants with adverse events [ Time Frame: 1 year after injections ]

such as death, heart failure, myocardial infarction, stroke, sustained/symptomatic ventricular tachycardia, bleeding, unplanned cardiovascular operation/reoperation and infection for one year.


Current Secondary Outcome:

  • Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation [ Time Frame: baseline, 24 weeks and 1 year ]
    as measured by serial echocardiograms and MRI scans.
  • Number of participants with incidence of mortality or need for transplantation after the BDCPA operation [ Time Frame: One year after injections ]
    Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
  • Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation [ Time Frame: 12 months post operative ]
    Changes in weight, height, and head circumference will be measured
  • Assessment of Co-morbidity [ Time Frame: up to 12 months follow-up ]
    Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.


Original Secondary Outcome:

  • Composite Change from baseline in right ventricular function, right ventricular end-diastolic volume, right ventricular end-systolic volume, right ventricular end-systolic diameter and tricuspid regurgitation [ Time Frame: baseline, 6 months, and 1 year ]
    as measured by serial echocardiograms and MRI scans.
  • Number of participants with incidence of mortality or need for transplantation after the BDCPA operation [ Time Frame: One year after injections ]
    Assessed through the number of participants with adverse events, hospitalizations, or transplantations.
  • Composite Changes in somatic growth velocity over time (weight, height, head circumference) from the BDCPA operation [ Time Frame: 12 months post operative ]
    Changes in weight, height, and head circumference will be measured
  • Assessment of Co-morbidity [ Time Frame: up to 12 months follow-up ]
    Assessment of cardiovascular mortality, all-cause morbidity, Cardiovascular morbidity, re-hospitalizations, need for transplantation.


Information By: University of Miami

Dates:
Date Received: December 24, 2014
Date Started: April 2015
Date Completion: October 2022
Last Updated: March 9, 2017
Last Verified: March 2017