The CHILD Trial: Hypoplastic Left Heart Syndrome Study.
Summary
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.
Timeline
- Start
- 2019-10-16
- Primary completion
- 2024-07-01
- Completion
- 2024-07-01
Outcome
Mixed primary results
paper The trial met its primary safety and feasibility endpoints. PMID 41258851 ↗
paper However, the primary efficacy endpoint-changes in RV size or function measured by cardiac magnetic resonance and echocardiography-was not achieved. PMID 41258851 ↗
Publications
- Kaushal S, Hare JM, Mahle WT, Khan A, Ohye RG, Slesnick TC, Chai PJ, Shashidharan S, Robinson JD, Jone PN, Doman T, Si MS, Lu JC, Bacallao K, Nettina AE, Lamazares R, Saltzman RG, Simpson LM, Li R, Bettencourt JE, Mansoor K, Davis BR, Deatrick KB, Yang J, Mishra R, Everett AD, Lai D, Davis ME. Phase I Randomized Study of Cardiac Stem Cells in Patients With Hypoplastic Left Heart Syndrome: The CHILD Trial. JACC Heart Fail. 2026 Jan;14(1):102723. doi: 10.1016/j.jchf.2025.102723. Epub 2025 Nov 19.
- Kaushal S, Hare JM, Shah AM, Pietris NP, Bettencourt JL, Piller LB, Khan A, Snyder A, Boyd RM, Abdullah M, Mishra R, Sharma S, Slesnick TC, Si MS, Chai PJ, Davis BR, Lai D, Davis ME, Mahle WT. Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study). Pediatr Cardiol. 2022 Oct;43(7):1481-1493. doi: 10.1007/s00246-022-02872-6. Epub 2022 Apr 8.
- Ali MK, Ichimura K, Spiekerkoetter E. Promising therapeutic approaches in pulmonary arterial hypertension. Curr Opin Pharmacol. 2021 Aug;59:127-139. doi: 10.1016/j.coph.2021.05.003. Epub 2021 Jun 30.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | c-kit+ cells | Unknown | 12500 cells/kg | Other |