Clinical trial NCT04925024: Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS): A Phase IIb Clinical Trial.
Official title: Evaluation of Lomecel-B™ Injection in Patients With Hypoplastic Left Heart Syndrome (HLHS) : A Phase IIb Clinical Trial.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Hypoplastic Left Heart Syndrome
Keywords: Hypoplastic Left Heart Syndrome
Pediatrics
Heart Defects
Congenital Cardiovascular Diseases
Stem Cells
Lead sponsor: Longeveron Inc.
Sponsor class: INDUSTRY
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Ann & Robert H Lurie Children's Hospital of Chicago
name: The University of Texas Health Science Center, Houston
First posted: 2021-06-14
Enrollment: 38
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion:

All participants must have HLHS (includes all types) requiring Stage II palliation (Glenn or Hemi-Fontan operation).

Exclusion:

1. Requirement for ongoing mechanical circulatory support immediately prior to Stage II palliation within 5 days
2. Need for concomitant surgery for aortic coarctation or tricuspid valve repair or Endocardial fibroelastosis (EFE) resection or left ventricle recruitment procedures
3. Undergoing the Stage I (Norwood) procedure that does not have HLHS
4. Serum positivity for: human immunodeficiency virus (HIV); hepatitis B virus surface antigen (HBV BsAg); and/or viremic hepatitis C virus (HCV). This criterion can be ascertained by one of three ways:

   1. Documented history of mother's testing conducted during pregnancy
   2. Documented history of participants testing.
   3. If above documentation is not available blood will be obtained from participant at Screening/Baseline.
5. Parent/guardian that is unwilling or unable to comply with necessary follow-up
6. Unsuitability for the study based on the Investigator's clinical opinion
7. Known hypersensitivity to dimethyl sulfoxide (DMSO)
8. Presence of a pacemaker, or anticipated placement of a pacemaker, at the time of the Stage II palliation

Primary outcomes:
measure: Change in right ventricular ejection fraction (RVEF)
timeFrame: Baseline, 12 Months
Secondary outcomes:
measure: Change in right ventricular ejection fraction (RVEF)
timeFrame: Baseline, 6 Months
measure: Change in right ventricular mass index at diastole
timeFrame: Baseline, 12 Months
measure: Change in right ventricular end-diastolic volume index (RVEDVI)
timeFrame: Baseline, 12 Months
measure: Change in right ventricular end-systolic volume index (RVESVI)
timeFrame: Baseline, 12 Months
measure: Change in right ventricular global longitudinal strain and strain rate
timeFrame: Baseline, 12 Months
measure: Change in right ventricular global circumferential strain and strain rate
timeFrame: Baseline, 12 Months
measure: Change in right atrial volume index
timeFrame: Baseline, 12 Months
measure: Change in right ventricular work index
timeFrame: Baseline, 12 Months
measure: Change in tricuspid regurgitation severity
timeFrame: Baseline, 12 Months
measure: Change in RV compliance/diastolic function
timeFrame: Baseline, 12 Months
measure: Change in weight
timeFrame: Baseline, 12 Months
measure: Change in length (height)
timeFrame: Baseline, 12 Months
measure: Change in head circumference
timeFrame: Baseline, 12 Months
measure: Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
timeFrame: Baseline, 12 Months
measure: Change in modified Ross Heart Failure Classification score
timeFrame: Baseline, 12 Months
measure: Change in PedsQL™ Infant Scales
timeFrame: Baseline, 12 Months
measure: Freedom from unplanned catheter intervention needed to address the pulmonary arteries or aorta.
timeFrame: Baseline, 12 Months
measure: Change in biomarkers/cytokines
timeFrame: Baseline, 12 Months
measure: Participants experiencing treatment emergent serious adverse events (TE-SAEs)
timeFrame: 30 days post Stage II palliation
measure: Participants experiencing major adverse cardiac events (MACE)
timeFrame: Baseline, 12 Months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Advocate Children's Hospital
state: Illinois
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Omaha
country: United States
facility: Children's Nebraska
state: Nebraska
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: UTHealth-McGovern Medical School
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital/University of Utah
state: Utah
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Stu Berger, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kaushal S, Hare JM, Hoffman JR, Boyd RM, Ramdas KN, Pietris N, Kutty S, Tweddell JS, Husain SA, Menon SC, Lambert LM, Danford DA, Kligerman SJ, Hibino N, Korutla L, Vallabhajosyula P, Campbell MJ, Khan A, Naioti E, Yousefi K, Mehranfard D, McClain-Moss L, Oliva AA, Davis ME. Intramyocardial cell-based therapy with Lomecel-B during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: the ELPIS phase I trial. Eur Heart J Open. 2023 Jan 11;3(2):oead002. doi: 10.1093/ehjopen/oead002. eCollection 2023 Mar.