Lomecel-B Delivered During Stage II Surgery for Hypoplastic Left Heart Syndrome (ELPIS)
- Conditions
- HLHS
- Registration Number
- NCT03525418
- Lead Sponsor
- Longeveron Inc.
- Brief 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
- Detailed 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 & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 10
all patients must have HLHS (all types) requiring BDCPA surgery.
all patients must not have any of the following.
- Significant coronary artery sinusoids.
- Requirement for mechanical circulatory support prior to BDCPA surgery.
- Underlying evidence of arrhythmia requiring anti-arrhythmia therapy.
- Need for concomitant surgery for aortic coarctation or tricuspid valve repair.
- HLHS and restrictive or intact atrial septum.
- Undergoing the Stage I (Norwood) procedure that does not have HLHS.
- Serum positivity for: HIV; hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV).
- Parent/guardian that is unwilling or unable to comply with necessary follow-up.
- Unsuitability for the study based on the Investigator's clinical opinion.
- Documented chromosomal abnormalities
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method 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. Evaluated through 1 year post-treatment. 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.
- Secondary Outcome Measures
Name Time Method Efficacy: Change from baseline in right ventricular ejection fraction (%). Evaluated through 1 year post-treatment. Used to assess cardiac function.
Efficacy: Change from baseline in right ventricular end-diastolic volume. Evaluated through 1 year post-treatment. Used to assess cardiac function.
Efficacy: Change in head circumference (in centimeters). Evaluated through 1 year post-treatment. Used to assess change in somatic growth.
Efficacy: Change from baseline in right ventricular end-diastolic diameter. Evaluated through 1 year post-treatment. Used to assess cardiac function.
Efficacy: Change from baseline tricuspid regurgitation. Evaluated through 1 year post-treatment. Used to assess cardiac function. Measured by serial echocardiograms and MRI.
Efficacy: Change from baseline in right ventricular end-systolic volume. Evaluated through 1 year post-treatment. Used to assess cardiac function.
Efficacy: Change in weight (in kilograms). Evaluated through 1 year post-treatment. Used to assess change in somatic growth.
Efficacy: Change in height (in centimeters). Evaluated through 1 year post-treatment. Used to assess change in somatic growth.
Efficacy: Number of patients with Treatment-Emergent Adverse Events, and total number of occurrences of Treatment-Emergent Adverse Events, through-out participation in trial. Evaluated through 1 year post-treatment. 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.
Trial Locations
- 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
University of Utah/Heart Center-Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Emory University/Childen's Healthcare of Atlanta🇺🇸Atlanta, Georgia, United States