Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome (ELPIS)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02398604
Recruitment Status : Terminated (Study was closed due to Sponsor switched to Longeveron. New Identifier is NTC03525418)
First Posted : March 25, 2015
Last Update Posted : June 25, 2019
Sponsor:
Collaborator:
University of Maryland, College Park
Information provided by (Responsible Party):
Joshua M Hare, University of Miami

Tracking Information
First Submitted Date  ICMJE December 24, 2014
First Posted Date  ICMJE March 25, 2015
Last Update Posted Date June 25, 2019
Study Start Date  ICMJE April 2015
Actual Primary Completion Date March 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2017)
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 Measures  ICMJE
 (submitted: March 24, 2015)
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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2017)
  • 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 Measures  ICMJE
 (submitted: March 24, 2015)
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome
Official Title  ICMJE 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 Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
20 patients and 10 patients will be controls with a total of 30 HLHS patients.
Masking: None (Open Label)
Masking Description:
HLHS patients which will be randomized to the treatment and control arms in a 1:1 ratio
Primary Purpose: Treatment
Condition  ICMJE Hypoplastic Left Heart Syndrome
Intervention  ICMJE
  • Drug: Allo-hMSCs
    Allogeneic Human Mesenchymal Stem Cells
    Other Name: stem cells; Allogeneic Human Mesenchymal Stem Cells
  • Drug: Placebo
    Placebo
    Other Name: Placebo comparator
Study Arms  ICMJE
  • Experimental: Group A - Allo-hMSCs
    Group A: Fifteen (20) patients will be treated with Allogenic human mesenchymal stem cells (Allo-hMSCs): A concentration of 5 million cells/ml delivered in a dose of 2.5 x 10^5 cells per kg of recipient (5 million/20kg) Allo-hMSCs. The entire dose of the cells will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation.
    Intervention: Drug: Allo-hMSCs
  • Placebo Comparator: Group B
    Group B: Fifteen (10) patients will be treated with a placebo comparator. The placebo will be divided and delivered in 6-10 open intramyocardial injections during the BDCPA operation.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: June 21, 2019)
5
Original Estimated Enrollment  ICMJE
 (submitted: March 24, 2015)
30
Actual Study Completion Date  ICMJE March 2019
Actual Primary Completion Date March 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Subjects with hypoplastic left heart syndrome (all types) requiring BDCPA surgery.

Exclusion Criteria:

  • Have HLHS and restrictive or intact atrial septum.
  • Be undergoing the Norwood procedure that do not have HLHS.
  • Have significant coronary artery sinusoids.
  • Require mechanical circulatory support prior to surgery.
  • Have an underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
  • Parent or guardian unwilling or unable to comply with necessary follow-up(s)
  • Be serum positive for HIV, hepatitis BsAg (B Surface Antigen) or viremic hepatitis C.
  • Be unsuitable for inclusion in the study, in the opinion of the investigator.
  • Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 30 Days   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02398604
Other Study ID Numbers  ICMJE 20140718
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Joshua M Hare, University of Miami
Study Sponsor  ICMJE Joshua M Hare
Collaborators  ICMJE University of Maryland, College Park
Investigators  ICMJE
Principal Investigator: Sunjay Kaushal, MD, Ph.D. University of Maryland, College Park
PRS Account University of Miami
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP