Clinical Trials

This phase III clinical trial is studying how well combination chemotherapy and surgery work in treating young patients with Bilateral Wilms tumor and children who are a special risk for forming tumors in both kidneys. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving combination chemotherapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving it after surgery may kill any tumor cells that remain after surgery.

The purpose of this study is to evaluate long-term safety and tolerability data in eligible CLCZ696B2319 (PANORAMA-HF) pediatric patients receiving open-label sacubitril/valsartan.

This randomized phase II/III trial studies how well pazopanib hydrochloride, combination chemotherapy, and radiation therapy work and compares it to radiation therapy alone or in combination with pazopanib hydrochloride or combination chemotherapy in treating patients with newly diagnosed non-rhabdomyosarcoma soft tissue sarcomas that can be removed by surgery. Radiation therapy uses high energy x-rays to kill tumor cells. Drugs used in chemotherapy, such as ifosfamide and doxorubicin hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. It is not yet known whether radiation therapy works better when given with or without combination chemotherapy and/or pazopanib hydrochloride in treating patients with non-rhabdomyosarcoma soft tissue sarcomas.

The purpose of this proposed study is to design a psychometrically valid measure of hearing handicap for the pediatric population.

The purpose of this study is to evaluate the pharmacokinetics (what the body does to the study medication) and safety of subcutaneously (under the skin) administered golimumab in pediatric participants (aged 2 to 17 years) with moderately to severely active Ulcerative Colitis (UC).

The purpose of this study is to conduct a comprehensive clinical and biomechanical screening of high school, collegiate-level, recreational, and Olympic/professional-level athletes with the goal of identifying individual functional and performance deficits that lead to future injury.

Congenital heart disease (CHD) is an abnormal formation that occurs during the development of a baby’s heart, heart valves and/or large vessels such as the aorta artery. CHD is the most common cause of major congenital defects accounting for almost 30% of all defects (Van der Linde D, JACC 2011). While the statistics vary among studies, the best birth prevalence estimate is 8 per 1000 live births (Bernier PL 2010). In the USA, CHD affects 1% of all births per year (Krasuki & Bashore 2016), with an estimated 40,000 babies born with any type of heart defect every year (Benjamin Emelia 2018).  Twenty-five percentof these are affected by a severe congenital heart defect (https://www.cdc.gov/ncbddd/heartdefects/data.html#References). Children with CHD who survive after the surgical procedures can develop heart failure and require a heart transplant at any time in their lives. Infants born with CHD need immediate medical attention and multiple follow-ups throughout their lives. Besides the social and economic impact of CHD on the individual and family lives, CHD treatment places a significant financial burden on the healthcare system. Simeone et al, (2014) reported that the cost of CHD hospitalization in the US was approximately $5.6 billion in 2019, accounting for 15.1% of the total cost for all pediatric hospitalizations in that year.

The important improvements in CHD diagnosis and surgical treatment in the last decades has led to an increased survival of newborns affected with heart defects. A large number of CHD can be diagnosed during pregnancy, and the patients can present a broad range of symptoms. Forms of CHD are usually classified based on their severity, from mild to severe. One of the mildest forms of CHD is atrial septal defect, which can be undetectable until adulthood (Hoffman & Kaplan, 2002) and VSD (Penny DJ, 2011). On the other hand, severe CHD that requires multiple palliative surgeries includes single ventricle defects, such as hypoplastic left heart syndrome (HLHS) and tricuspid atresia.

The survival of infants with CHD will depend on the severity of the defect and the time of diagnosis and treatment received. The one-year survival of newborns with severe or critical CHD (generally any type of surgery/procedures in their first year of life) is estimated to be 75%.

Stem cell therapy has emerged as a new paradigm of treatment in the field of CHD with promising results. Cardiac regeneration has been the focus of acquired, adult heart disease for many years. However, congenital heart disease with structural abnormalities may also be a good target for other research studies. In fact, the pediatric heart is naturally growing and may be amendable to regenerative strategies. Furthermore, the initial pre-clinical and clinical studies have demonstrated that the delivery of stem cells into the heart of patients with CHD is feasible and safe. Moreover, the cell therapy approach, along with the standard surgical palliation, seems to offer benefits over surgical treatment alone. Even though the number of cell therapy clinical trials for CHD has increased in the last decade, more long-term follow-up studies are needed in this population setting in order to define the role of stem cell therapy in the clinical practice. Therefore, confirming our ability to produce autologous cells (cells from the patient's own body) from patients with severe CHD is an important step towards the long-term goal of being able to discover innovative cell-based protocols.

The purpose of this study is to determine whether speech recognition in noise can be improved by the addition of a special listening program to the sound processor. Thus we propose to assess speech perception in noise for pediatric cochlear implant patients using both their everyday listening program as well an autosensitivity program.

This Is a Humanitarian Use Protocol for the Impella RP® System which is indicated for providing circulatory assistance for up to 14 days in pediatric or adult patients with a body surface area ≥ 1.5 m2 who develop acute right heart failure or decompensation following left ventricular assist device implantation, myocardial infarction, heart transplant, or open-heart surgery. 

This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of Hypoplastic Left Heart Syndrome (HLHS) children undergoing a scheduled Glenn surgical procedure. The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.