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

HLHS Clinical Trial

— ELPIS  

Official title:

Lomecel-B Injection in Patients With Hypoplastic Left Heart Syndrome: A Phase I/II Study (ELPIS)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03525418
• Other study ID #: 00-0000-05
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: February 21, 2018
• Est. completion date: December 31, 2025

Study information

• Verified date: October 2023
• Source: Longeveron Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: December 31, 2025
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 1 Year

Eligibility

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

Related Conditions & MeSH terms

• HLHS
• Hypoplastic Left Heart Syndrome

Intervention

Biological:
• Longeveron Mesenchymal Stem Cells
Allogeneic bone marrow-derived mesenchymal stem cell

Locations

Country	Name	City	State
United States	Emory University/Childen's Healthcare of Atlanta	Atlanta	Georgia
United States	Johns Hopkins University Hospital	Baltimore	Maryland
United States	University of Maryland Medical Center	Baltimore	Maryland
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	University of Utah/Heart Center-Primary Children's Hospital	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Longeveron Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change from baseline in right ventricular ejection fraction (%).	Used to assess cardiac function.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change from baseline in right ventricular end-systolic volume.	Used to assess cardiac function.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change from baseline in right ventricular end-diastolic volume.	Used to assess cardiac function.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change from baseline in right ventricular end-diastolic diameter.	Used to assess cardiac function.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change from baseline tricuspid regurgitation.	Used to assess cardiac function. Measured by serial echocardiograms and MRI.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change in weight (in kilograms).	Used to assess change in somatic growth.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change in height (in centimeters).	Used to assess change in somatic growth.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Change in head circumference (in centimeters).	Used to assess change in somatic growth.	Evaluated through 1 year post-treatment.
Secondary	Efficacy: Number of patients with Treatment-Emergent Adverse Events, and total number of occurrences of Treatment-Emergent Adverse Events, through-out participation in trial.	Treatment-Emergent Adverse Events will be assessed via incidence of co-morbidity, which include: cardiovascular morbidity; need for transplantation; re-hospitalizations; cardiovascular mortality; and all-cause mortality.	Evaluated through 1 year post-treatment.