Clinical Trials

The purpose of this study is to assess how well glembatumumab vedotin works in treating patients with osteosarcoma that has come back (recurrent) or does not respond to treatment (refractory). Monoclonal antibodies, such as glembatumumab vedotin, may find tumor cells and help kill them.

The purpose of this study is to test the safety and effectiveness of Covered CP Stents to repair or prevent aortic wall injury associated with Coarctation of the Aorta (CoA). Coarctation of the aorta (CoA) is a congenital abnormality producing obstruction to blood flow through the aorta. Coarctation can occur in isolation, in association with bicuspid aortic valve or with major cardiac malformations. CoA accounts for 5-8% of the 8/1000 (4-6/10,000) children born with congenital heart disease. Most CoA is newly diagnosed in childhood; < 25% recognized beyond 10 yrs. CoA is mostly repaired in childhood by surgery or by balloon catheter dilation. Recurrence rates range from 5-20%. Recurrence is often not recognized until adolescence. Balloon expandable stents have become the predominant therapy in the USA and Europe for CoA treatment in this age group. There are no FDA approved stents for this use. Biliary stents are currently being used off label. Enrollment into a trial of bare metal Cheatham Platinum (CP) Stents, designed for use in CoA, is completed. The Coarctation of the Aorta Stent Trial (COAST) aims to confirm safety and efficacy of CP Stent for native and recurrent CoA. There are CoA patients with clinical situations that place them at high risk of aortic wall injury during bare metal stenting. Extreme narrowing, genetic aortic wall weakness and advanced age are examples. Patients may present with aortic wall injury (aneurysm) related to prior CoA repair. The occurrence after surgical repair is 3-4% and after balloon dilation 10-20%. Repair of these aneurysms is surgically challenging. The use of fabric-covered CP Stents to prevent or repair aortic wall injury has become the treatment of choice in Europe and recently in the US through the FDA Compassionate Use process. There are no alternative devices available in the US.

This phase III trial studies the side effects and how well risk-based therapy works in treating younger patients with newly diagnosed liver cancer. Surgery, chemotherapy drugs (cancer fighting medicines), and when necessary liver transplant are the main current treatments for hepatoblastoma. The stage of the cancer is one factor used to decide the best treatment. Treating patients according to the risk group they are in may help get rid of the cancer, keep it from coming back, and decrease the side effects of chemotherapy.

Background: Sometimes the cancer advanced alveolar soft part sarcoma (ASPS) cannot be helped with surgery or other treatment. The drug atezolizumab unblocks the immune system. This allows immune cells to recognize and attack tumor cells. The drug could shrink cancer but could also have side effects. Researchers want to study if the drug will shrink a tumor in people with advanced ASPS. Objective: To test good and bad effects of the drug atezolizumab. Eligibility: People at least 6 years old with ASPS that cannot be cured with surgery Design: Participants will be screened with heart and pregnancy tests. Some may have scans or other tests. At the study start, participants will have: - Medical history - Physical exam - Heart, blood, and pregnancy tests - Scan to measure tumor - Optional tumor sample taken Each study cycle is 21 days. In cycle 1: - On day 1, participants will have blood tests. They will get the study drug in a vein for about 1 hour. They will be observed for a few hours after. - On days 8 and 15, participants will have blood tests. In other cycles: - On day 1, participants will get the study drug in a vein for about 1 hour. - On days 1 and 15, participants will have blood tests. Some participants will have a tumor sample taken in cycle 3. Participants will have tumor scans at the end of cycle 3 and then every 2 cycles. Participants can stay in the study as long as their cancer does not get worse. Participants will be observed for 30 days after stopping the study drug.

The objective of this study is to evaluate the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) delivered into the myocardium of the right ventricle of subjects with Ebstein anomaly undergoing surgical Ebstein repair. Additionally, the potential cardiovascular benefits will also be evaluated. This add-on procedure is anticipated to pose little risk to the subject and has the potential to foster a new strategy that leverages the regenerative capacity of individuals with congenital heart disease during the surgically mandated Ebstein repair.

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 offer ovarian tissue cryopreservation to female pediatric patients (birth-17 years of age) with fertility threatening medical diagnoses or facing surgery, chemotherapy or radiation therapy that may cause loss of reproductive potential. Since this procedure is currently considered experimental, the establishment of an IRB protocol under which this opportunity can be offered is needed.

The purpose of this study is to evaluate how children and young adults perceive their midline sternotomy scars (in terms of appearance, associated symptoms, consciousness, satisfaction with appearance/symptoms, and impact on quality of life)? 

The purpose of this study is to confirm detection of the H3K27M mutation in patient biofluids (CSF, blood, urine, saliva) and uncover the molecular histone (alkaline protein) profile of H3K trimethylation and H3S phosphorylation in the circulating nucleosomes isolated from the biofluids of patients with H3K27M tumors.

This study is being done to learn about cataract surgery in children. The study involves the collection of information about children who have had cataract surgery. This information will include when the child developed cataract, how the cataract has been treated, problems that may have occurred related to surgery, and whether anyone else in the family has had a cataract. Doctors throughout the United States and possibly other parts of the world are taking part in the study. We hope that gathering the information from many children we can improve the care of children with cataracts.