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

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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

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.

Condition or disease Intervention/treatment Phase
Hypoplastic Left Heart Syndrome Drug: Allo-hMSCs Drug: Placebo Phase 1

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
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
Official Title: Allogeneic Human MEsenchymal Stem Cell (hMSC) Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
Study Start Date : April 2015
Actual Primary Completion Date : March 2019
Actual Study Completion Date : March 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.
Drug: Allo-hMSCs
Allogeneic Human Mesenchymal Stem Cells
Other Name: stem cells; Allogeneic Human Mesenchymal Stem Cells

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.
Drug: Placebo
Placebo
Other Name: Placebo comparator




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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

  4. 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.



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Ages Eligible for Study:   up to 30 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT02398604


Locations
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United States, Maryland
University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Sponsors and Collaborators
Joshua M Hare
University of Maryland, College Park
Investigators
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Principal Investigator: Sunjay Kaushal, MD, Ph.D. University of Maryland, College Park

Additional Information:
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Responsible Party: Joshua M Hare, Sponsor, University of Miami
ClinicalTrials.gov Identifier: NCT02398604     History of Changes
Other Study ID Numbers: 20140718
First Posted: March 25, 2015    Key Record Dates
Last Update Posted: June 25, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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:
Pediatrics
HLHS
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