Clinical Trial: Mesoblast Stem Cell Therapy for Patients With Single Ventricle and Borderline Left Ventricle
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: Mesoblast Stem Cell Therapy for Patients With Single Ventricle and Borderline Left Ventricle
Brief Summary:
Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment following bidirectional Glenn (BDG) or undergoing BDG with plans for LV recruitment will be considered for enrollment in this study. Those patients enrolled in the study will be randomized to either the experimental arm or control arm of the study. Those patients randomized to the experimental arm will receive mesenchymal precursor cells (MPCs) injected directly into the LV endocardium during their LV recruitment or BDG procedure. Those patients randomized to the control arm will receive normal standard of care during their procedure with no injection of MPCs.
It is believed that injection of MPCs will help improve the chances of those patients with single ventricle or borderline left ventricle being converted to biventricular circulation which could improve quality of life and longevity over palliation.
Detailed Summary:
This is a prospective, single center, safety and feasibility, blinded, randomized trial to evaluate the use of MPCs in children with complex cardiac anatomy requiring surgical repair. Patients scheduled to undergo bidirectional Glenn (BDG) with future plans for LV recruitment, or patients with a history of BDG who are currently scheduled to undergo LV recruitment will be eligible.
Twenty-four subjects will be enrolled, 12 to the MPC treatment arm and 12 to the control arm following a 1:1 randomization schema. Randomization will be stratified according to surgeon to assure random distribution of subjects by surgeon. Families, the biostatistician, all clinical staff outside the operating room, and research staff completing data analysis will be blinded to randomization assignment. The PI, operating room staff, and research staff assisting with delivery will be unblinded to the randomization assignment. Families will be made aware of their randomization assignment at the completion of the study on Visit 4, following completion of all scheduled assessments. Those randomized to the treatment arm will receive MPCs injected directly into the LV endocardium following clinical surgical maneuvers to recruit the LV (mitral valve repair, aortic valve repair, and/or resection of endocardial fibroelastosis) or BDG. Those subjects randomized to the control arm will receive standard LV recruitment or BDG with no injection.
All cardiac tissue acquired as part of a clinically indicated procedure will be collected on enrolled subjects. Most of the tissue sample will be analyzed at the time of collection (histology and H+E stain); the remainder will be banked for potential future testing.
Sponsor: Boston Children’s Hospital
Current Primary Outcome: Safety- Incidence of severe adverse events [ Time Frame: 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Safety- Absence of PRA status change or local inflammation [ Time Frame: 12 months ]
Original Secondary Outcome: Safety- Absence of PRA status change or local inflammation [ Time Frame: 12 months ]
Information By: Boston Children’s Hospital
Dates:
Date Received: March 8, 2017
Date Started: April 2017
Date Completion: February 2021
Last Updated: March 15, 2017
Last Verified: March 2017
