TY - JOUR
T1 - Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study)
AU - Kaushal, Sunjay
AU - Hare, Joshua M.
AU - Shah, Aakash M.
AU - Pietris, Nicholas P.
AU - Bettencourt, Judith L.
AU - Piller, Linda B.
AU - Khan, Aisha
AU - Snyder, Abigail
AU - Boyd, Riley M.
AU - Abdullah, Mohamed
AU - Mishra, Rachana
AU - Sharma, Sudhish
AU - Slesnick, Timothy C.
AU - Si, Ming Sing
AU - Chai, Paul J.
AU - Davis, Barry R.
AU - Lai, Dejian
AU - Davis, Michael E.
AU - Mahle, William T.
N1 - Publisher Copyright:
© 2022, The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.
PY - 2022/10
Y1 - 2022/10
N2 - 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 Registrationhttps://clinicaltrials.gov/ct2/home NCT03406884, First posted January 23, 2018.
AB - 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 Registrationhttps://clinicaltrials.gov/ct2/home NCT03406884, First posted January 23, 2018.
KW - Cardiac stem cells
KW - Cardiac-derived progenitor cells
KW - Hypoplastic left heart syndrome
UR - http://www.scopus.com/inward/record.url?scp=85127709363&partnerID=8YFLogxK
U2 - 10.1007/s00246-022-02872-6
DO - 10.1007/s00246-022-02872-6
M3 - Article
AN - SCOPUS:85127709363
SN - 0172-0643
VL - 43
SP - 1481
EP - 1493
JO - Pediatric Cardiology
JF - Pediatric Cardiology
IS - 7
ER -