The CHILD Trial
Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome: An Open Label Pilot Study
What is HLHS?
Congenital heart defects or diseases are problems with the heart’s structure that are present at birth, which may change the normal flow of blood through the heart. Congenital heart defects are the most common type of birth defects. Hypoplastic left heart syndrome (HLHS) is a complex and rare defect at birth (congenital). In HLHS, the left side of the heart is critically underdeveloped. In a newborn with HLHS, the left side of the heart can't effectively pump blood to the body therefore, the right side of the heart must pump blood to the lungs and the rest of the body.
For further information: https://www.cdc.gov/ncbddd/heartdefects/hlhs.html
Due to advances in medical care, the outlook for newborn babies with HLHS is better now than in the past. Experiments performed in small and large animals suggest that giving c-kit+ cells that can repair damaged heart tissues and improve the ability of diseased hearts to pump blood throughout the body.
The purpose of this study is to test how safe and effective autologous ckit+ cell therapy is for managing HLHS. It is hoped that by injecting these stem cells into the right ventricle of the heart, that there will be better right heart pumping function as the child gets older.
Primary Objective: To overlay a novel cell therapeutic strategy on the two-stage surgical procedures that HLHS patients typically undergo in the first year of life:
- Stage I Norwood operation in the neonatal period
- Stage II BDCPA / GLENN procedure operation at approximately 4 months of age
To accomplish this, c-kit+ cells will be grown from heart tissue collected during the Stage I Norwood operation. Usually, this tissue is discarded after the surgery, however for this trial, it will be collected and shipped to the University of Miami Interdisciplinary Stem Cell Institute. There the tissue will be processed, cultured, and expanded to grow c-kit+ cells. The culture and expansion process can take up to 8 to 10 weeks. Upon completion, c-kit+ cells will be cryopreserved and tested for quantity, quality and safety as mandated by FDA for an Investigational New Drug (IND). Upon completion of these tests, cells will be shipped back to the clinical center for administration back to the patient during the Stage II BDCPA / GLENN Procedure operation.
Follow-up visits occur at the following timepoints after study product administration: 5 days, 4 weeks, 6 months, and 12 months.
The CHILD trial is the first time autologous cardiac stem cells will be injected into patients diagnosed with HLHS. However, extensive research has previously shown the potential benefits of using cardiac stem cells as a treatment therapy.
Click on the articles listed below to find out about the latest findings on cardiac stem cell characterization and stem cell therapy treatment in patients with HLHS.