Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome

Phase 2 Active not recruiting 95 enrolled Timothy J Nelson, MD, PhD Children's Hospital Colorado · collab Children's Hospital Los Angeles · collab Children's Hospital Medical Center, Cincinnati · collab Children's Hospital of Philadelphia · collab Children's Hospitals and Clinics of Minnesota · collab Children's of Alabama · collab Ochsner Health System · collab University of Oklahoma · collab

Non-randomizedParallel-groupOpen-labelTreatment

Summary

Researchers want to better understand what happens to the heart when the stem cells are injected directly into the muscle of the right side of the heart during the Stage II palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS) or HLHS variant. Researchers want to see if there are changes in the heart's structure/function following this stem cell-based therapy and compared to children that have not had cell-based therapy.

Timeline

Start: 2019-06-06
Primary completion: 2022-05-22
Completion: 2026-02

Outcome

Missed primary endpoint

paper unfavorable change in longitudinal cardiac strain in the treatment group compared with an improvement in strain in the control group (unadjusted p =.032) PMID 40312733 ↗

paper failed to enhance cardiac function in patients with hypoplastic left heart syndrome. PMID 40312733 ↗

Publications

Gallego-Navarro C, Jaggers J, Burkhart HM, Carlo WF, Morales DL, Qureshi MY, Rossano JW, Hagen CE, Seisler DK, Peral SC, Nelson TJ. Autologous umbilical cord blood mononuclear cell therapy for hypoplastic left heart syndrome: a nonrandomized control trial of the efficacy and safety of intramyocardial injections. Stem Cell Res Ther. 2025 May 1;16(1):215. doi: 10.1186/s13287-025-04316-3.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	UCB-MNCs	Cell therapy	1e+06 cells/kg	Other
Subject	UCB-MNCs	Cell therapy	3e+06 cells/kg	Other