Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial. (ELPIS II)

Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS) : A Phase IIb Clinical Trial.

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Hypoplastic Left Heart Syndrome
• Intervention / Treatment: Biological: Lomecel-B medicinal signaling cells

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital of Los Angeles
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Chicago, Illinois, United States, 60453
      • Advocate Children's Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Nebraska
    • Omaha, Nebraska, United States, 68114
      • Children's Nebraska
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital/University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 1 year (Child)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in right ventricular ejection fraction (RVEF)	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.	Baseline, 12 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in right ventricular ejection fraction (RVEF)	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.	Baseline, 6 Months
Change in right ventricular mass index at diastole	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.	Baseline, 12 Months
Change in right ventricular end-diastolic volume index (RVEDVI)	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.	Baseline, 12 Months
Change in right ventricular end-systolic volume index (RVESVI)	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.	Baseline, 12 Months
Change in right ventricular global longitudinal strain and strain rate	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.	Baseline, 12 Months
Change in right ventricular global circumferential strain and strain rate	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.	Baseline, 12 Months
Change in right atrial volume index	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.	Baseline, 12 Months
Change in tricuspid regurgitation severity	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.	Baseline, 12 Months
Change in weight	Efficacy measured as change in participant's weight in kg and will be measured serially to assess change in somatic growth.	Baseline, 12 Months
Change in length (height)	Efficacy measured as change in participant's length (height) in cm and will be measured serially to assess change in somatic growth.	Baseline, 12 Months
Change in head circumference	Efficacy measured as change in participant's head circumference in cm and will be measured serially to assess change in somatic growth.	Baseline, 12 Months
Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)	Efficacy measured as change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) in pg/ml and will be measured serially by blood draw.	Baseline, 12 Months
Change in modified Ross Heart Failure Classification score	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).	Baseline, 12 Months
Change in PedsQL™ Infant Scales	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.	Baseline, 12 Months
Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta.	Efficacy measured as the number and percentage of participants who do not undergo unplanned catheter interventions to address pulmonary arteries or the aorta.	Baseline, 12 Months
Change in biomarkers/cytokines	Efficacy measured as change in biomarkers/cytokines in pg/ml and/or ng/ml and will be measured serially by blood draw.	Baseline, 12 Months
Participants experiencing treatment emergent serious adverse events (TE-SAEs)	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: all-cause mortality; 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; sepsis; need for new permanent pacemaker; stroke or embolic event to the brain	30 days post Stage II palliation
Participants experiencing major adverse cardiac events (MACE)	Safety will be reported as the number of participants with adjudicated events including worsening heart failure; listed for heart transplant; heart failure hospitalization; cardiovascular mortality	Baseline, 12 Months

Collaborators and Investigators

• Sponsor: Longeveron Inc.
• Collaborators: The University of Texas Health Science Center, Houston; National Heart, Lung, and Blood Institute (NHLBI); Ann & Robert H Lurie Children's Hospital of Chicago
• Investigators: Principal Investigator: Stu Berger, MD, Ann & Robert H Lurie Children's hospital of Chicago

Publications and helpful links

General Publications

• Kaushal S, Hare JM, Hoffman JR, Boyd RM, Ramdas KN, Pietris N, Kutty S, Tweddell JS, Husain SA, Menon SC, Lambert LM, Danford DA, Kligerman SJ, Hibino N, Korutla L, Vallabhajosyula P, Campbell MJ, Khan A, Naioti E, Yousefi K, Mehranfard D, McClain-Moss L, Oliva AA, Davis ME. Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: the ELPIS phase I trial. Eur Heart J Open. 2023 Jan 11;3(2):oead002. doi: 10.1093/ehjopen/oead002. eCollection 2023 Mar.

Helpful Links

• ELPIS II study website
• Earlier phase I study (ELPIS)

Study record dates

Study Major Dates

• Study Start (Actual): June 25, 2021
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): August 31, 2026

Study Registration Dates

• First Submitted: June 8, 2021
• First Submitted That Met QC Criteria: June 8, 2021
• First Posted (Actual): June 14, 2021

Study Record Updates

• Last Update Posted (Estimated): June 25, 2025
• Last Update Submitted That Met QC Criteria: June 24, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Stem Cells; Pediatrics; Hypoplastic Left Heart Syndrome; Heart Defects; Congenital Cardiovascular Diseases
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Pathologic Processes; Heart Diseases; Disease; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Syndrome; Hypoplastic Left Heart Syndrome
• Other Study ID Numbers: 2021-4531; 7UG3HL148318-02 (U.S. NIH Grant/Contract); 1U24HL148316-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No