Phase I Amount
$1,683,381
One of the most complex forms of congenital heart disease, hypoplastic left heart syndrome (HLHS), affects an average of 1 in every 5,000 newborns with a mortality rate of up to 35 percent during the first year of life. The overall goal f our research is to develop safe and effective ways to improve short-term and long-term survival rates in babies born with HLHS. We aim to introduce a novel cell-based therapeutic strategy in the three-stage surgical procedure that HLHS patients typically undergo. Our hypothesis is that injected allogeneic cardiosphere derived cells (CDCs) will boost and regenerate the myocardium of the single right ventricle in HLHS patients, improving right ventricular function and, thus, short and long-term clinical outcomes. The aims of this Phase I/2 clinical trials are to evaluate the feasibility and safety of direct intra-coronary injection of allogeneic CDCs in these HLHS patients at the time of cauterization for their second operation. This proposal will translate a series of basic and pre-clinical observations into a potentially paradigm-shifting therapy to meet an unmet medical need in HLHS patients. In addition, this study will bring a deeper insight into the biology of stem cells in human patients which have never been studied before in pediatric congenital heart patients.
Public Health Relevance Statement: Public Health Relevance: Hypoplastic left heart syndrome is one of the most complex forms of congenital heart disease, with a reported incidence of 9% of children born with congenital heart disease. Despite these strides in medical care during the last twenty years, the mortality rate of these infants remain at 25-35% during the first year of life because mostly of right ventricle dysfunction. This proposal will translate a series of basic and pre-clinical observations into a potentially paradigm-shifting cell based therapy to improve the right ventricular function and thus improve clinical outcomes.
Project Terms: Adult; Affect; Age-Months; Age-Years; Allogenic; Arrhythmia; base; Blinded; Blood Circulation; California; Cardiac; Cardiac development; Caring; Cauterize; Cell Therapy; Cells; Cessation of life; Child; Childhood; Clinical; Clinical Protocols; Clinical Research; clinical research site; Clinical trial protocol document; Clinical Trials; Complex; congenital heart disorder; Coronary; Data; Diagnosis; Dose; Echocardiography; Event; experience; Family suidae; Fibrosis; Fontan Procedure; Functional disorder; Funding; Goals; Heart; Heart failure; Human; Hypoplastic Left Heart Syndrome; imaging modality; Immune; improved; improved functioning; improved outcome; Incidence; Infant; Infusion procedures; Injection of therapeutic agent; insight; Institutes; Institutional Review Boards; Investigational Drugs; laboratory facility; Life; Live Birth; Los Angeles; Magnetic Resonance; manufacturing facility; Maryland; Medical; Medical center; meetings; Modeling; mortality; Myocardial; Myocardial Infarction; Myocardium; Natural regeneration; Neonatal; Newborn Infant; non-invasive imaging; novel; novel therapeutics; operation; Operative Surgical Procedures; Outcome; Outcome Measure; palliation; patient population; Patients; pediatric patients; Phase; Positioning Attribute; pre-clinical; pressure; prevent; Protocols documentation; public health relevance; Randomized; Regenerative Medicine; Reporting; Research; Research Personnel; Right Ventricular Function; Right ventricular structure; Safety; Secure; Series; Staging; stem cell biology; stem cell technology; Stem cells; Survival Rate; Therapeutic; Time; Translating; Transplantation; Tricuspid Valve Insufficiency; tumor; United States National Institutes of Health; Universities
