This study is for patients that have been diagnosed with rhabdomyosarcoma (RMS). The investigational drug in this study is Temsirolimus. The purpose of this study is to find out if we can improve the treatment for subjects with intermediate risk RMS by adding temsirolimus to VAC/IV therapy. Participants can expect to be in this study for approximately 1 year and would like to continue to follow-up with the patient every year for about 10 years.
This study is for patients that have been diagnosed with High Risk B-Lymphoblastic Leukemia (HR B-ALL). The investigational drug in this study is Ruxolitinib. The purpose of this study is to find out if the study drug, ruxolitinib, in combination with standard HR B-ALL treatment is safe and effective in children, adolescents, and young adults with HR B-ALL. Participants can expect to be in this study for the treatment period of approximately 26 months (females) or 38 months (males) plus the post-treatment follow-up. Subjects are considered on study during the post-treatment follow-up period until the subject is deceased, lost to follow-up, or until the study is completed. Subjects in this study will be followed until all enrolled subjects have been followed for 3 years from Day 1 or are deceased or lost to follow-up.
This study is for patients that have been diagnosed with Central Nervous System (CNS) Tumors. The investigational drugs in this study are Nivolumab and Ipilimumab. The purpose of this study is to test the effectiveness, safety, and tolerability of Nivolumab when given alone or when combined with Ipilimumab. The duration of patient participation may be more than 3 years. If enrolled in treatment, the exact length of time will depend on the patients response to treatment.
This study is for pediatric patients that have been diagnosed with cancer and are receiving chemotherapy. The investigational drugs in this study are netupitant and palonosetron. The purpose of this study is to learn more about how well the combination of oral netupitant and oral palonosetron works in preventing nausea and vomiting associated with chemotherapy in children. Participation in the study will last for a maximum of 31 days, which includes a screening period up to 14 days before randomization (up to 7 days for patients aged less than 2 years), the day of enrollment/randomization, administration of study drugs and chemotherapy (Study Day 1), and the control visits (Study Days 2 to 5).
In this study, TB-403 is a reviewed as a possible treatment for relapsed or refractory medulloblastoma (MB), neuroblastoma (NB), Ewing sarcoma (ES), or alveolar rhabdomyosarcoma (ARMS). This study will be conducted to determine the maximum tolerated dose (MTD), or the appropriate dosage, of TB-403. TB-403 will be given during a 28-day dose period in pediatric subjects ages greater than 6 months to less than 18 years of age. Study subjects require a diagnoses and/or history of relapsed or refractory medulloblastoma (MB), neuroblastoma (NB), Ewing sarcoma (ES), or alveolar rhabdomyosarcoma (ARMS). The study will also assess safety and tolerability of TB-403 as well as the preliminary effictiveness for use in pediatric patients with MB, NB, ES or ARMS.
High risk Neuroblastoma (NB) remains a challenge in pediatric oncology, accounting for 15% of all pediatric cancer deaths. While most patients are able to attain remission, approximately 50% will relapse. Once relapsed, there is currently no curative treatment for these children, and for these children the 5-year survival rate is <10%. As such, new therapeutic approaches are needed to treat these children.
These more aggressive forms of NB respond poorly to hormonal and chemotherapeutic approaches, and therefore, there is a great need for antineoplastic agents with novel mechanisms of action. The MYCN protein up-regulates ornithine decarboxylase (ODC), a gene encoding for the ODC enzyme that is pivotal in polyamine biosynthesis. High polyamine content and elevated ODC activities are commonly found in NB as well as many other tumors, and therefore, suppression of polyamines in cancer cells is an effective means to reduce tumor cell proliferation. We have shown ODC inhibition reverses the LIN28/Let7 pathway, an important pathway in cell differentiation and regulation of glycolytic metabolism. In studying this pathway, it was found to be regulated as well by the NFkB pathway. We have therefore studied the combination of DFMO with Bortezomib and showing synergy of these medications in neuroblastoma in vitro and in vivo. This study will address this concept in children with relapsed or refractory neuroblastoma.
The purpose of this study is to test the feasibility (ability to be done) of an experimental test called the "Pediatric Gene Analysis Platform," to help plan cancer treatment. This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology is based on genetic testing done at the Translational Genomics Institute (TGen) in Phoenix, Arizona and methods of genetic analysis created by NMTRC and TGen. The Pediatric Gene Analysis Platform is being used to discover new ways to understand pediatric cancers and potentially predict the best treatments for patients with cancer in the future. This experimental technology has not been approved by the U.S. Food and Drug Administration. This study plan is not studying the effectiveness of the proposed combinations of therapy.
The overall goal of this study is to find out what effects, good and/or bad, a low
dose and a high dose of lenalidomide have on children, adolescents and young
adults with recurrent (has come back after being treated), refractory (has not gone
away with previous treatment), or progressive (is not responding to previous
treatments) Juvenile Pilocytic Astrocytomas (JPA) and Optic Pathway Gliomas
Although JMML is an uncommon disease, it occurs exclusively in very young
children (median age ~ 2.5 years) indicating an increased risk for TRM and late
effects associated with maximum intensity conditioning regimens. Moreover, there is currently no agreed upon standard of care preparative regimen in use for
patients with JMML. Previous studies suggest that there are significant toxicities
associated with conditioning regimens currently in use today. Moreover, the
relapse rates were fairly high and it appears that further escalation of the
conditioning regimens is unlikely to produce significant improvements in EFS or
relapse rates without unacceptable TRM. Therefore, it is essential that novel
strategies be developed to reduce the high rates of relapse that have been
the United States, it is standard treatment for patients with high-risk neuroblastoma (NBL) to receive the drugs carboplatin, etoposide and melphalan (CEM) as the preparative regimen in Consolidation therapy prior to Autologous Stem Cell Transplant (ASCT). BuMel Consolidation therapy has recently been studied in patients with high-risk NBL in some European countries. The findings from those studies indicate that the use of BuMel prior to ASCT may be linked to an increase in the survival rate for patients when compared to CEM. Those studies also indicate that the chance of the disease coming back (a relapse) may be lower among the patients who received BuMel Consolidation therapy. In North America the BuMel combination is considered experimental. In this study, researchers want to find out if a combination of busulfan and melphalan (BuMel) can be given as Consolidation therapy prior to ASCT for subjects with newly diagnosed high-risk NBL. The main goal of this study is to find out what effects, good and/or bad, a BuMel preparative regimen given before ASCT has on people with newly diagnosed high-risk NBL.