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08-04-2022 | Original Article

Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study)

Authors: Sunjay Kaushal, Joshua M. Hare, Aakash M. Shah, Nicholas P. Pietris, Judith L. Bettencourt, Linda B. Piller, Aisha Khan, Abigail Snyder, Riley M. Boyd, Mohamed Abdullah, Rachana Mishra, Sudhish Sharma, Timothy C. Slesnick, Ming-Sing Si, Paul J. Chai, Barry R. Davis, Dejian Lai, Michael E. Davis, William T. Mahle

Published in: Pediatric Cardiology | Issue 7/2022

Abstract

Mortality in infants with hypoplastic left heart syndrome (HLHS) is strongly correlated with right ventricle (RV) dysfunction. Cell therapy has demonstrated potential improvements of RV dysfunction in animal models related to HLHS, and neonatal human derived c-kit⁺ cardiac-derived progenitor cells (CPCs) show superior efficacy when compared to adult human cardiac-derived CPCs (aCPCs). Neonatal CPCs (nCPCs) have yet to be investigated in humans. The CHILD trial (Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome) is a Phase I/II trial aimed at investigating intramyocardial administration of autologous nCPCs in HLHS infants by assessing the feasibility, safety, and potential efficacy of CPC therapy. Using an open-label, multicenter design, CHILD investigates nCPC safety and feasibility in the first enrollment group (Group A/Phase I). In the second enrollment group, CHILD uses a randomized, double-blinded, multicenter design (Group B/Phase II), to assess nCPC efficacy based on RV functional and structural characteristics. The study plans to enroll 32 patients across 4 institutions: Group A will enroll 10 patients, and Group B will enroll 22 patients. CHILD will provide important insights into the therapeutic potential of nCPCs in patients with HLHS.

Clinical Trial Registration https://clinicaltrials.gov/ct2/home NCT03406884, First posted January 23, 2018.

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Title: Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study)
Authors: Sunjay Kaushal
Joshua M. Hare
Aakash M. Shah
Nicholas P. Pietris
Judith L. Bettencourt
Linda B. Piller
Aisha Khan
Abigail Snyder
Riley M. Boyd
Mohamed Abdullah
Rachana Mishra
Sudhish Sharma
Timothy C. Slesnick
Ming-Sing Si
Paul J. Chai
Barry R. Davis
Dejian Lai
Michael E. Davis
William T. Mahle
Publication date: 08-04-2022
Publisher: Springer US
Published in: Pediatric Cardiology / Issue 7/2022
Print ISSN: 0172-0643
Electronic ISSN: 1432-1971
DOI: https://doi.org/10.1007/s00246-022-02872-6

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