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

L

Longeveron

Status and phase

Active, not recruiting
Phase 2
Phase 1

Conditions

HLHS

Treatments

Biological: Longeveron Mesenchymal Stem Cells

Study type

Interventional

Funder types

Industry

Identifiers

NCT03525418
00-0000-05

Details and patient eligibility

About

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

Full description

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).

Enrollment

10 patients

Sex

All

Ages

1 day to 1 year old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Double Blind

10 participants in 3 patient groups

Cohort A - Phase 1 (Open Label)
Experimental group
Description:
10 consecutive HLHS patients will be enrolled and treated with Longeveron Mesenchymal Stem Cells (LMSCs). A single administration of LMSCs will be performed via intramyocardial injections during the Stage II (BDCPA) surgery. Dosing is based on body weight. Each LMSC-treated patient will be given 2.5 x 105 LMSCs per kg of body weight. The entire dose of the cells will be roughly 600 microliters.
Treatment:
Biological: Longeveron Mesenchymal Stem Cells
Cohort B - Phase 2 Treatment Group
Experimental group
Description:
Double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Longeveron Mesenchymal Stem Cells (LMSCs) (Cohort B, 10 patients) performed via intramyocardial injections during the Stage II (BDCPA) surgery, or will receive no cells and no injection (Cohort C, 10 patients) during the Stage II (BDCPA) surgery. The second stage is to obtain preliminary safety and efficacy data the will enable and guide a subsequent larger Phase 2 trial.
Treatment:
Biological: Longeveron Mesenchymal Stem Cells
Cohort C - Phase 2 Control Group
No Intervention group
Description:
Double-blinded, in which 20 HLHS patients will be randomized to either receive treatment with Longeveron Mesenchymal Stem Cells (LMSCs) (Cohort B, 10 patients) performed via intramyocardial injections during the Stage II (BDCPA) surgery, or will receive no cells and no injection (Cohort C, 10 patients) during the Stage II (BDCPA) surgery. The second stage is to obtain preliminary safety and efficacy data the will enable and guide a subsequent larger Phase 2 trial.

Trial contacts and locations

5

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Data sourced from clinicaltrials.gov

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