Stem Cell Therapy for Single Ventricle Heart Conditions

Palo Alto (17 mi)

Overseen bySitaram M Emani, MD

Age: < 18

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Boston Children's Hospital

No Placebo Group

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for children under 5 with heart conditions like hypoplastic left heart syndrome, undergoing specific heart procedures. It's not for kids who've had myocardial tumors, certain valve issues, severe arrhythmias, allergies to DMSO or animal products, previous stem cell therapy for the heart, or recent other cell therapies.

Treatment Details

The study tests if injecting mesenchymal precursor cells (MPCs) into the left ventricle during surgery can help convert single-ventricle hearts to two-chamber hearts. Kids will be randomly assigned to get MPCs or just standard care without MPCs during their procedure.

2Treatment groups

Experimental Treatment

Active Control

Group I: Treatment ArmExperimental Treatment1 Intervention

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. MPCs will be delivered directly into the LV endocardium via a 23-25 gauge needle following completion of all surgical procedures. A total dose of 20 million cells will be delivered, divided evenly into \~11 injections of 50 µL each. The total volume is not to exceed 2.0 mL.

Group II: Control ArmActive Control1 Intervention

Those subjects randomized to the control arm will receive standard LV recruitment or BDG with no injection.