Lomecel-B Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS)

Longeveron Inc.10 enrolled

Overview

This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS. Lomecel-B will be delivered via intramyocardial injections

This study is designed to assess the safety, tolerability, and efficacy of Lomecel-B (formerly LMSCs) as an adjunct therapy to the standard stage II (BDCPA) surgical intervention for HLHS, which is typically performed at 4 - 6 months after birth. Lomecel-B will be delivered via intramyocardial injections. A total of 30 patients will be enrolled in 2 stages with 3 Cohorts. In the first stage, 10 consecutive HLHS patients will be enrolled and treated with Lomecel-B (Cohort A). The first 3 patients will be treated no less than 5 days apart, and will be evaluated for any treatment-emergent adverse events (TE-AEs) (e.g., induced myocardial infarction or perforation). These patients will undergo full evaluation for 5 days to demonstrate safety prior to proceeding with the remainder of the cohort. After 6 months post-treatment of the last patient of Cohort A, a formal safety review will be conducted prior to proceeding to the next phase. The second stage is double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Lomecel-B (Cohort B, 10 patients), or will receive no cells and no injection (Cohort C, 10 patients).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

HLHS

Interventions

Longeveron Mesenchymal Stem CellsBIOLOGICAL

Allogeneic bone marrow-derived mesenchymal stem cell

Eligibility

Sex: ALLMin age: 1 DayMax age: 1 Year

Medical Language ↔ Plain English

Inclusion Criteria: all patients must have HLHS (all types) requiring BDCPA surgery. Exclusion Criteria: all patients must not have any of the following. 1. Significant coronary artery sinusoids. 2. Requirement for mechanical circulatory support prior to BDCPA surgery. 3. Underlying evidence of arrhythmia requiring anti-arrhythmia therapy. 4. Need for concomitant surgery for aortic coarctation or tricuspid valve repair. 5. HLHS and restrictive or intact atrial septum. 6. Undergoing the Stage I (Norwood) procedure that does not have HLHS. 7. Serum positivity for: HIV; hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV). 8. Parent/guardian that is unwilling or unable to comply with necessary follow-up. 9. Unsuitability for the study based on the Investigator's clinical opinion. 10. Documented chromosomal abnormalities

Locations (5)

Emory University/Childen's Healthcare of Atlanta

Atlanta, Georgia, United States

University of Maryland Medical Center

Baltimore, Maryland, United States

Johns Hopkins University Hospital

Baltimore, Maryland, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Safety: To evaluate the safety and feasibility of intramyocardial injection of LMSCs during the Stage II (BDCPA) operation for HLHS via incidence of Treatment-Emergent Serious Adverse Events.

The incidence of Treatment-Emergent Serious Adverse Events will be evaluated, including: sustained/symptomatic ventricular tachycardia requiring intervention with inotropic support; aggravation of heart failure; myocardial infarction; unplanned cardiovascular operation for cardiac tamponade; infection during the first month post-treatment; and death.