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

Longeveron

Status and phase: Active, not recruiting; Phase 2

Conditions: Hypoplastic Left Heart Syndrome

Treatments: Biological: Lomecel-B medicinal signaling cells

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT04925024

2021-4531

7UG3HL148318-02 (U.S. NIH Grant/Contract)

1U24HL148316-01A1 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Enrollment: 40 patients

Sex: All

Ages: Under 12 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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40 participants in 2 patient groups

Lomecel B Group

Experimental group

Description:

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

Treatment:

Biological: Lomecel-B medicinal signaling cells

No Study Intervention Control Group

No Intervention group

Description:

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