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

Purpose

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

  • Hypoplastic Left Heart Syndrome

Eligibility

Eligible Ages
Under 12 Months
Eligible Genders
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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
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.

Recruiting Locations

UTHealth-McGovern Medical School
Houston, Texas 77030
Contact:
Rebecca Sam
713-500-5746
Rebecca.M.Sam@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
Longeveron Inc.

Study Contact

Stephanie Brazis, MPH
312-227-0201
sbrazis@luriechildrens.org