Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial.
- Conditions
- Hypoplastic Left Heart Syndrome
- Interventions
- Biological: Lomecel-B medicinal signaling cells
- Registration Number
- NCT04925024
- Lead Sponsor
- Longeveron Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 38
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Lomecel B Group Lomecel-B medicinal signaling cells Participants randomized to receive Lomecel-B injections during their Stage II palliation.
- Primary Outcome Measures
Name Time Method Change in right ventricular ejection fraction (RVEF) Baseline, 12 Months Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
- Secondary Outcome Measures
Name Time Method Change in right ventricular ejection fraction (RVEF) Baseline, 6 Months Efficacy will be reported as change in right ventricular ejection fraction (RVEF) assessed as a percentage and will be measured via cardiac magnetic resonance (CMR) imaging.
Change in right ventricular end-systolic volume index (RVESVI) Baseline, 12 Months Efficacy will be reported as change in right ventricular end systolic volume index (RVESVI) assessed as ml/m\^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in right ventricular global longitudinal strain and strain rate Baseline, 12 Months Efficacy will be reported as change in right ventricular global longitudinal strain and strain rate assessed as % and s\^-1 respectively and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in tricuspid regurgitation severity Baseline, 12 Months Efficacy will be reported as change in the categorical qualitative assessment of tricuspid valve regurgitation (trivial, mild, moderate, severe) and will be measured via transthoracic echocardiography.
Change in right ventricular mass index at diastole Baseline, 12 Months Efficacy will be reported as change in right ventricular mass index at diastole assessed as g/m\^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in PedsQL™ Infant Scales Baseline, 12 Months The PedsQL™ Infant Scales is a generic health related quality of life instrument specifically for healthy and ill infants ages 1-24 months. Efficacy measured as change in PedsQL total improvement score and will be measured serially by parental proxy-report. Higher scores are indicative of better quality of life.
Change in biomarkers/cytokines Baseline, 12 Months Efficacy measured as change in biomarkers/cytokines in pg/ml and/or ng/ml and will be measured serially by blood draw.
Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta. Baseline, 12 Months Efficacy measured as the number and percentage of participants who do not undergo unplanned catheter interventions to address pulmonary arteries or the aorta.
Participants experiencing treatment emergent serious adverse events (TE-SAEs) 30 days post Stage II palliation Safety will be reported as the number of participants experiencing treatment emergent serious adverse events (TE-SAEs) assessed by treating physician within the 30 days following study procedure. These include: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention; cardiogenic shock; unplanned cardiovascular operation for bleeding due to right ventricular intramyocardial injection site bleeding in the first five days after stage II palliation; worsening of cardiac function; local infection or systemic infection; need for new permanent pacemaker; death.
Change in right atrial volume index Baseline, 12 Months Efficacy will be reported as the change in right atrial volume index as ml/m\^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in weight Baseline, 12 Months Efficacy measured as change in participant's weight in kg and will be measured serially to assess change in somatic growth.
Change in head circumference Baseline, 12 Months Efficacy measured as change in participant's head circumference in cm and will be measured serially to assess change in somatic growth.
Participants experiencing major adverse cardiac events (MACE) Baseline, 12 Months Safety will be reported as the number of participants with adjudicated events including cardiovascular morbidity; need for transplantation; re-hospitalizations; cardiovascular mortality; all-cause mortality.
Change in right ventricular end-diastolic volume index (RVEDVI) Baseline, 12 Months Efficacy will be reported as change in right ventricular end diastolic volume index (RVEDVI) assessed as ml/m\^2 and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in right ventricular global circumferential strain and strain rate Baseline, 12 Months Efficacy will be reported as the change in right ventricular global circumferential strain and strain rate as % and s\^-1 respectively, and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in right ventricular work index Baseline, 12 Months Efficacy will be reported as the change in right ventricular work index as (mmHg x mL x beats) / min and will be measured via serial cardiac magnetic resonance (CMR) imaging.
Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) Baseline, 12 Months Efficacy measured as change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP) in pg/ml and will be measured serially by blood draw.
Change in RV compliance/diastolic function Baseline, 12 Months Efficacy will be reported as change in diastolic function as measured by the tricuspid E/E' ratio, evaluating the tricuspid inflow E and A velocities and ratio, and tricuspid annular DTI E'A' velocities. Function reported as normal diastolic function (no abnormalities in these measures), mildly impaired diastolic function (1 abnormal measure), moderately impaired diastolic function (2 abnormal measures), and severely impaired diastolic function (3 abnormal measures). These measures will be measured via transthoracic echocardiography.
Change in length (height) Baseline, 12 Months Efficacy measured as change in participant's length (height) in cm and will be measured serially to assess change in somatic growth.
Change in modified Ross Heart Failure Classification score Baseline, 12 Months The Ross Heart Failure Classification provides a global assessment of heart failure severity in infants. Efficacy measured as change in classification and will be measured serially via physician's assessment using the modified Ross Heart Failure Classification; classifications defined as Class 1 (no limitations of physical activity); Class 2 (may experience symptoms during moderate exercise but not during rest); Class 3 (symptoms with minimal exertion that interfere with normal daily activity); Class 4 (unable to carry out physical activity/has symptoms at rest that worsen with exertion).
Trial Locations
- Locations (12)
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Advocate Children's Hospital
🇺🇸Chicago, Illinois, United States
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Nebraska
🇺🇸Omaha, Nebraska, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
UTHealth-McGovern Medical School
🇺🇸Houston, Texas, United States
Primary Children's Hospital/University of Utah
🇺🇸Salt Lake City, Utah, United States