​Children's Heart Center

Research & Clinical Trials

What Are Clinical Trials?

Clinical trials are research studies that help find new and better ways to prevent, diagnose and treat disease. Almost every treatment available to patients today was first tested in a clinical trial. Clinical trials are how we find new therapies, improve current treatments, and reduce current side effects. When you or your child participate in a clinical trial, you are actively participating in improving outcomes and finding new treatments. 

HLHS Consortium Member

As a member of the HLHS Consortium, The Children’s Hospital at OU Medicine is one of six institutions across the country dedicated to discovering and developing more effective methods to manage and treat hypoplastic left heart syndrome (HLHS)

Established by Mayo Clinic in Rochester, Minn., the HLHS Consortium is a means to study more HLHS patients, collecting a greater volume of knowledge than would be possible otherwise. More patients mean more opportunities to discover new avenues for treatment, care and enhanced quality of life for the long term.

Led by Harold Burkhart, M.D., head of the pediatric cardiothoracic surgery team at OU Medicine, membership in the Hypoplastic Left Heart Syndrome Consortium establishes Oklahoma as a regional center for families to receive care closer to home, helping create new efficiencies, share best practices and define protocols that improve lives of children and families affected by HLHS.

Nationwide, Hypoplastic Left Heart Syndrome Consortium members include: Mayo Clinic, Children's Hospital of Philadelphia, Children's Hospital of Los Angeles, Children's Minnesota, Children's Hospital Colorado, and The Children's Hospital at OU Medicine. The collaboration is expanding the development of cell-based, innovative research opportunities to transform the lives of people living with hypoplastic left heart syndrome. Consortium partners contribute not only financial resources, but the time and expertise of highly skilled specialists. 

The HLHS Consortium was established through the philanthropy of the Todd and Karen Wanek Family Program at Mayo, founded in 2010. The program is responsible for funding clinical trials that have yielded significant results, changing lives for the better.

The Children's Hospital at Dusk

    What is HLHS?

    HLHS is a congenital (present at birth) heart disease, affecting about 1,000 newborns in the United States each year. It causes critical underdevelopment of the left side of the heart, placing the full burden of heart function on the right side. While HLHS is the most severe congenital heart defect that occurs in infants, it is so rare that research initiatives and development of new therapies have been limited.

    Until recently, the course of treatment for HLHS has included drug therapy to prevent closure of the connection between the heart’s right and left sides, followed by corrective surgery or transplant. Surgical treatment is actually a series of three staged reconstructive procedures.  

    • The first (Norwood) typically occurs within days of birth.
    • The second procedure (Glenn) follows in the next three to six months.
    • The final surgery (Fontan) is usually performed at 2 to 4 years of age.

    A phase I clinical trial recently conducted by consortium partners was the first such research monitored by the Food and Drug Administration. The study demonstrated the potential of regenerative therapy for HLHS through the collection, processing and direct injection of an infant’s own stem cells into the heart during a surgical procedure. The injected stem cells serve to strengthen the heart muscle.

    Advantages of a Consortium

    Among other advantages, the consortium promotes complementary, cross-functional teams that are able to gain and share expertise more rapidly. Through the consortium, progress is accelerated and positive outcomes are being realized. Consortium participants may also have the capacity to provide local or regional care that minimizes the burden of travel for patients’ families.