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Trial record 1 of 6 for:    Lomecel-B
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Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial. (ELPISII)

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: NCT04925024
Recruitment Status : Recruiting
First Posted : June 14, 2021
Last Update Posted : August 12, 2022
Sponsor:
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
The University of Texas Health Science Center, Houston
Longeveron Inc.
Information provided by (Responsible Party):
Sunjay Kaushal, Ann & Robert H Lurie Children's Hospital of Chicago

Brief Summary:
The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B. Lomecel-B contains human mesenchymal stem cells (MSCs) as the active ingredient. MSCs are special cells in the body that are able to change into other types of cells, such as heart, blood, and muscle cells. MSCs are found in various tissues of the body, such as the bone marrow, which is the spongy tissue inside of your bones. Lomecel-B uses MSCs from bone marrow of unrelated young healthy donors. These are called "allogeneic", and do not require donor matching to the patient.

Condition or disease Intervention/treatment Phase
Hypoplastic Left Heart Syndrome Biological: Lomecel-B medicinal signaling cells Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase IIb Clinical Trial.
Actual Study Start Date : June 25, 2021
Estimated Primary Completion Date : March 2025
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lomecel B Group
Participants randomized to receive Lomecel-B injections during their Stage II palliation.
Biological: Lomecel-B medicinal signaling cells
A single administration of Lomecel-B will be performed via 6-10 intramyocardial injections into the right ventricle during the participant's standard of care stage II palliation. Dosing is based on body weight. Each patient will be given 2.5 x 10^5 cells per kg of body weight. The entire dose of the cells will be roughly 600 microliters.

No Intervention: No Study Intervention Control Group
Participants randomized to receive no study intervention during their Stage II palliation.



Primary Outcome Measures :
  1. Change in right ventricular ejection fraction (RVEF) [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.


Secondary Outcome Measures :
  1. Change in right ventricular ejection fraction (RVEF) [ Time Frame: Baseline, 6 Months ]
    Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.

  2. Change in right ventricular mass index at diastole [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in right ventricular mass index at diastole assessed as g/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  3. Change in right ventricular end-diastolic volume index (RVEDVI) [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in right ventricular end diastolic volume index (RVEDVI) assessed as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  4. Change in right ventricular end-systolic volume index (RVESVI) [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in right ventricular end systolic volume index (RVESVI) assessed as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  5. Change in right ventricular global longitudinal strain and strain rate [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in right ventricular global longitudinal strain and strain rate assessed as % and s^-1 respectively and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  6. Change in right ventricular global circumferential strain and strain rate [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as the change in right ventricular global circumferential strain and strain rate as % and s^-1 respectively, and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  7. Change in right atrial volume index [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as the change in right atrial volume index as ml/m^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  8. Change in right ventricular work index [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as the change in right ventricular work index as (mmHg x mL x beats) / min and will be measured via serial cardiac magnetic resonance (CMR) imaging.

  9. Change in tricuspid regurgitation severity [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in the categorical qualitative assessment of tricuspid valve regurgitation (trivial, mild, moderate, severe) and will be measured via transthoracic echocardiography.

  10. Change in RV compliance/diastolic function [ Time Frame: Baseline, 12 Months ]
    Efficacy will be reported as change in diastolic function as measured by the tricuspid E/E' ratio, evaluating the tricuspid inflow E and A velocities and ratio, and tricuspid annular DTI E'A' velocities. Function reported as normal diastolic function (no abnormalities in these measures), mildly impaired diastolic function (1 abnormal measure), moderately impaired diastolic function (2 abnormal measures), and severely impaired diastolic function (3 abnormal measures). These measures will be measured via transthoracic echocardiography.

  11. Change in weight [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as change in participant's weight in kg and will be measured serially to assess change in somatic growth.

  12. Change in length (height) [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as change in participant's length (height) in cm and will be measured serially to assess change in somatic growth.

  13. Change in head circumference [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as change in participant's head circumference in cm and will be measured serially to assess change in somatic growth.

  14. Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) in pg/ml and will be measured serially by blood draw.

  15. Change in modified Ross Heart Failure Classification score [ Time Frame: Baseline, 12 Months ]
    The Ross Heart Failure Classification provides a global assessment of heart failure severity in infants. Efficacy measured as change in classification and will be measured serially via physician's assessment using the modified Ross Heart Failure Classification; classifications defined as Class 1 (no limitations of physical activity); Class 2 (may experience symptoms during moderate exercise but not during rest); Class 3 (symptoms with minimal exertion that interfere with normal daily activity); Class 4 (unable to carry out physical activity/has symptoms at rest that worsen with exertion).

  16. Change in PedsQL™ Infant Scales [ Time Frame: Baseline, 12 Months ]
    The PedsQL™ Infant Scales is a generic health related quality of life instrument specifically for healthy and ill infants ages 1-24 months. Efficacy measured as change in PedsQL total improvement score and will be measured serially by parental proxy-report. Higher scores are indicative of better quality of life.

  17. Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta. [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as the number and percentage of participants who do not undergo unplanned catheter interventions to address pulmonary arteries or the aorta.

  18. Change in biomarkers/cytokines [ Time Frame: Baseline, 12 Months ]
    Efficacy measured as change in biomarkers/cytokines in pg/ml and/or ng/ml and will be measured serially by blood draw.

  19. Participants experiencing treatment emergent serious adverse events (TE-SAEs) [ Time Frame: 30 days post Stage II palliation ]
    Safety will be reported as the number of participants experiencing treatment emergent serious adverse events (TE-SAEs) assessed by treating physician within the 30 days following study procedure. These include: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention; cardiogenic shock; unplanned cardiovascular operation for bleeding due to right ventricular intramyocardial injection site bleeding in the first five days after stage II palliation; worsening of cardiac function; local infection or systemic infection; need for new permanent pacemaker; death.

  20. Participants experiencing major adverse cardiac events (MACE) [ Time Frame: Baseline, 12 Months ]
    Safety will be reported as the number of participants with adjudicated events including cardiovascular morbidity; need for transplantation; re-hospitalizations; cardiovascular mortality; all-cause mortality.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion:

All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).

Exclusion:

  1. Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days
  2. Need for concomitant surgery for aortic coarctation or tricuspid valve repair or Endocardial fibroelastosis (EFE) resection or left ventricle recruitment procedures
  3. Undergoing the Stage I (Norwood) procedure that does not have HLHS
  4. Serum positivity for: human immunodeficiency virus (HIV); hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV). This criterion can be ascertained by one of three ways:

    1. Documented history of mother's testing conducted during pregnancy
    2. Documented history of participants testing.
    3. If above documentation is not available blood will be obtained from participant at Screening/Baseline.
  5. Parent/guardian that is unwilling or unable to comply with necessary follow-up
  6. Unsuitability for the study based on the Investigator's clinical opinion
  7. Known hypersensitivity to dimethyl sulfoxide (DMSO)
  8. Presence of a pacemaker, or anticipated placement of a pacemaker, at the time of the Stage II palliation

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): NCT04925024


Contacts
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Contact: Kathleen Van't Hof 312-227-1549 KVantHof@luriechildrens.org
Contact: Shelly Sayre 713-500-9529 Shelly.L.Sayre@uth.tmc.edu

Locations
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United States, California
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Carly Weaver    323-361-8631    cweaver@chla.usc.edu   
Contact: Mike Gawad    323-361-3313    mgawad@chla.usc.edu   
Principal Investigator: Ram Subramanyan, MD, PhD         
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Emily Klingman, MSN, RN    404-785-7153    emily.klingman@choa.org   
Contact: Emily Elston    404-785-0535    emily.elston@choa.org   
Principal Investigator: William Mahle, MD         
United States, Illinois
Advocate Children's Hospital Recruiting
Chicago, Illinois, United States, 60453
Contact: Bonnie Hughes, RN/BSN/CCRC    708-296-2214    bonnie.hughes@aah.org   
Contact: Mary Murray, RN/BSN/CCRC    708-684-4576    mary.murray@aah.org   
Principal Investigator: Narutoshi Hibino, MD         
Principal Investigator: Rohit Loomba, MD         
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Carmen Diaz-Leos    312-227-5395    cdiazleos@luriechildrens.org   
Contact: Stephanie Brazis    312-227-0201    sbrazis@luriechildrens.org   
Principal Investigator: Sunjay Kaushal, MD, PhD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Breanna Piekarski, RN, MPH    617-919-4457    Breanna.Piekarski@cardio.chboston.org   
Principal Investigator: Sitaram Emani, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Kenneth Mucheck, MPH    513-803-0320    Kenneth.Mucheck@cchmc.org   
Contact: Sarah Speed    513-803-7022    Sarah.Speed@cchmc.org   
Principal Investigator: David Morales, MD         
United States, Utah
Primary Children's Hospital/University of Utah Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Linda Lambert, ARPN    801-587-7523    Linda.lambert@hsc.utah.edu   
Principal Investigator: Adil Husain, MD         
Principal Investigator: Edem Binka, MD         
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
National Heart, Lung, and Blood Institute (NHLBI)
The University of Texas Health Science Center, Houston
Longeveron Inc.
Investigators
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Principal Investigator: Sunjay Kaushal, MD, PhD Ann & Robert H Lurie Children's Hospital of Chicago
Additional Information:
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Responsible Party: Sunjay Kaushal, Principal Investigator, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT04925024    
Other Study ID Numbers: 2021-4531
7UG3HL148318-02 ( U.S. NIH Grant/Contract )
1U24HL148316-01A1 ( U.S. NIH Grant/Contract )
First Posted: June 14, 2021    Key Record Dates
Last Update Posted: August 12, 2022
Last Verified: August 2022
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 Sunjay Kaushal, Ann & Robert H Lurie Children's Hospital of Chicago:
Hypoplastic Left Heart Syndrome
Pediatrics
Heart Defects
Congenital Cardiovascular Diseases
Stem Cells
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