Clinical Trials

Despite recent improvements in outcome for children with newly diagnosed high-risk neuroblastoma, cure rates  remain  unsatisfactory.Further, these gains have been the result of interventions during the Consolidation (tandem autologous stem cell transplant) and Post-Consolidation (dinutuximab immunotherapy) phases of treatment, while rates of disease control during Induction have not improved in recent COG trials. The current phase 3 trial seeks to improve the event-free survival (EFS) for children with high-risk neuroblastoma through early integration of promising novel targeted therapies: targeted radiopharmaceutical therapy with 131I-MIBG or the ALK inhibitor, crizotinib. After enrollment, patients will receive one cycle of Induction chemotherapy. Subsequent therapy will be based upon MIBG avidity and ALK status. Patients with MIBG-avid, ALK wild type (or ALK unknown) disease will be randomized to one of three arms: A) current COG recommended high-risk therapy including four more cycles of Induction chemotherapy and surgical resection of the primary tumor, Consolidation with tandem transplant and focal external beam radiation, and dinutuximab immunotherapy with isotretinoin; B) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle; or C) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle and substitution of busulfan / melphalan (BuMel) single autologous stem cell transplant in place of tandem transplant. Patients with MIBG non-avid, ALK wild type (or ALK unknown) disease will be non-randomly assigned to receive current COG recommended high-risk therapy without the addition of 131I-MIBG. Patients with ALK aberrant tumors (ALK tyrosine kinase mutation or ALK amplification) will be non-randomly assigned to receive crizotinib added to current COG recommended high-risk therapy. The primary endpoint is EFS and 774 eligible and evaluable patients are anticipated to enroll over approximately 5 years. Key secondary endpoints are toxicity, end-Induction response, and overall survival. Late effects of therapy including targeted therapies will be compared with late effects of current COG recommended treatments Embedded correlative studies seek to understand predictors of benefit and resistance to 131I-MIBG and crizotinib.

The purpose of this study is to evaluate the challenges, behavioral patterns, and preferences of minority patient participation in clinical trials. Also, to develop and validate a personalized clinical trial educational platform to boost participation among underserved cancer patients.

The purpose of this study is to determine prevalence and incidence of osteopenia, osteoporosis, and fractures in patients with adenomas based on hormonal sub-type, and to determine the effect of abnormal steroid metabolome on bone density, bone metabolism, and fractures.

The purpose of this study is to create a research registry to prospectively collect research biospecimens and corresponding clinical data from subjects with an undiagnosed tumor or undifferentiated mass.

The purpose of this research is to follow people with adrenal disorders in order to make conclusions about the natural history of a particular adrenal disease as well as effect of various therapies and interventions decided on by you and your medical team. In addition, we will collect biomaterial from you at times you are being evaluated which will be used to discover novel biomarkers which can potentially improve the accuracy of current diagnostic tests and affect the management of patients with adrenal disorders. We will also include a control group (without known adrenal disease) to compare to the volunteers with adrenal disorders.