Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial.
ELPIS II
3 other identifiers
interventional
40
1 country
10
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2021
Longer than P75 for phase_2
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 8, 2021
CompletedFirst Posted
Study publicly available on registry
June 14, 2021
CompletedStudy Start
First participant enrolled
June 25, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2026
June 25, 2025
June 1, 2025
5 years
June 8, 2021
June 24, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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 Outcomes (18)
Change in right ventricular ejection fraction (RVEF)
Baseline, 6 Months
Change in right ventricular mass index at diastole
Baseline, 12 Months
Change in right ventricular end-diastolic volume index (RVEDVI)
Baseline, 12 Months
Change in right ventricular end-systolic volume index (RVESVI)
Baseline, 12 Months
Change in right ventricular global longitudinal strain and strain rate
Baseline, 12 Months
- +13 more secondary outcomes
Study Arms (2)
Lomecel B Group
EXPERIMENTALParticipants randomized to receive Lomecel-B injections during their Stage II palliation.
No Study Intervention Control Group
NO INTERVENTIONParticipants randomized to receive no study intervention during their Stage II palliation.
Interventions
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.
Eligibility Criteria
You may qualify if:
- All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Advocate Children's Hospital
Chicago, Illinois, 60453, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Nebraska
Omaha, Nebraska, 68114, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Primary Children's Hospital/University of Utah
Salt Lake City, Utah, 84113, United States
Related Publications (1)
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.
PMID: 36950450BACKGROUND
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Stu Berger, MD
Ann & Robert H Lurie Children's Hospital of Chicago
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 8, 2021
First Posted
June 14, 2021
Study Start
June 25, 2021
Primary Completion (Estimated)
June 30, 2026
Study Completion (Estimated)
August 31, 2026
Last Updated
June 25, 2025
Record last verified: 2025-06
Data Sharing
- IPD Sharing
- Will not share