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NCT Number: NCT04925024

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hypoplastic Left Heart Syndrome Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Heart Defects, Congenital Heart Diseases

Age range

Up to 12 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Children's Hospital of Los Angeles, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion:

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

Exclusion:

  • Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days
  • Need for concomitant surgery for aortic coarctation or tricuspid valve repair or Endocardial fibroelastosis (EFE) resection or left ventricle recruitment procedures
  • Undergoing the Stage I (Norwood) procedure that does not have HLHS
  • 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:
  • Documented history of mother's testing conducted during pregnancy
  • Documented history of participants testing.
  • If above documentation is not available blood will be obtained from participant at Screening/Baseline.
  • Parent/guardian that is unwilling or unable to comply with necessary follow-up
  • Unsuitability for the study based on the Investigator's clinical opinion
  • Known hypersensitivity to dimethyl sulfoxide (DMSO)
  • Presence of a pacemaker, or anticipated placement of a pacemaker, at the time of the Stage II palliation

Treatment and study plan

Lomecel-B medicinal signaling cells

Biological

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.

Primary outcomes

  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 outcomes

  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 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.

  9. 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.

  10. 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.

  11. 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.

  12. 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.

  13. 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).

  14. 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.

  15. 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.

  16. 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.

  17. 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: 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

  18. 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 worsening heart failure; listed for heart transplant; heart failure hospitalization; cardiovascular mortality

Sponsors and collaborators

Lead sponsor

Longeveron Inc.

Industry

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

Registry information

Official study title

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

Acronym: ELPIS II

Important dates

Study start
2021
Primary completion
2026
Study completion
2026
First posted
Jun 14, 2021
Registry last updated
Jun 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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