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Intramyocardial Cell Delivery: Observations in Murine Hearts
Published on: January 24, 2014
Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome (CHILD Study)
Sunjay Kaushal1, Joshua M Hare2, Aakash M Shah3
1Division of Cardiovascular-Thoracic Surgery, Ann & Robert H. Lurie Children's Hospital of Chicago, 225 E. Chicago Avenue, Chicago, IL, 60611, USA. skaushal@luriechildrens.org.
Insights
The CHILD trial investigates using neonatal cardiac progenitor cells (CPCs) to treat hypoplastic left heart syndrome (HLHS) in infants. This study assesses the safety and effectiveness of this novel cell therapy for improving heart function.
Area of Science:
- Cardiology
- Regenerative Medicine
- Pediatric Heart Surgery
Background:
- Infant mortality in hypoplastic left heart syndrome (HLHS) is linked to right ventricle (RV) dysfunction.
- Cell therapy shows promise for RV dysfunction in HLHS animal models.
- Neonatal cardiac progenitor cells (CPCs) are more effective than adult CPCs in preclinical studies.
Purpose of the Study:
- To evaluate the feasibility, safety, and potential efficacy of intramyocardial injection of autologous neonatal CPCs (nCPCs) in infants with HLHS.
- To assess nCPC therapy in a Phase I/II clinical trial (the CHILD trial).
Main Methods:
- The CHILD trial is an open-label, multicenter Phase I/II study.
- Group A (Phase I) assesses safety and feasibility in 10 patients.
- Group B (Phase II) is a randomized, double-blinded, multicenter trial assessing efficacy in 22 patients, focusing on RV function and structure.
Main Results:
- This abstract presents the design of the CHILD trial, not interim or final results.
- The trial aims to enroll 32 patients across 4 institutions.
Conclusions:
- The CHILD trial is expected to provide critical insights into the therapeutic potential of nCPCs for HLHS.
- This research may pave the way for new treatments for infants with this complex congenital heart defect.
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.
