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

NCT ID: NCT04925024

Last Updated: 2025-06-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-25

Study Completion Date

2026-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hypoplastic Left Heart Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Lomecel B Group

Participants randomized to receive Lomecel-B injections during their Stage II palliation.

Group Type EXPERIMENTAL

Lomecel-B medicinal signaling cells

Intervention Type 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.

No Study Intervention Control Group

Participants randomized to receive no study intervention during their Stage II palliation.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

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
Maximum Eligible Age

12 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role collaborator

Longeveron Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Stu Berger, MD

Role: PRINCIPAL_INVESTIGATOR

Ann & Robert H Lurie Children's Hospital of Chicago

Locations

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Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Advocate Children's Hospital

Chicago, Illinois, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Children's Nebraska

Omaha, Nebraska, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Primary Children's Hospital/University of Utah

Salt Lake City, Utah, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 36950450 (View on PubMed)

Related Links

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http://www.elpistrial.org

ELPIS II study website

Other Identifiers

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7UG3HL148318-02

Identifier Type: NIH

Identifier Source: secondary_id

View Link

1U24HL148316-01A1

Identifier Type: NIH

Identifier Source: secondary_id

View Link

2021-4531

Identifier Type: -

Identifier Source: org_study_id

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