View clinical trials related to Neuroblastoma.
Filter by:Metaiodobenzylguanidine (MIBG) is a substance that is taken up by neuroblastoma cells. MIBG is combined with radioactive iodine (131 I) in the laboratory to form a radioactive compound 131 I-MIBG. This radioactive compound delivers radiation specifically to the cancer cells and causes them to die. The purpose of this research protocol provides a mechanism to deliver MIBG therapy when clinically indicated, but also to provide a mechanism to continue to collect efficacy and toxicity data that will be provided. A recent New Approaches to Neuroblastoma Therapy (NANT) phase 2 randomized trial of 131I-MIBG with or without radiation sensitizers for relapsed refractory or persistent neuroblastoma enrolled 114 patients ages 1-30 years showed that Arm A (MIBG alone) had a response rate of 17%, Arm B (MIBG with Vincristine and Irnotecan) had a response rate of 14% and Arm C (MIBG with vorinostat) had a response rate of 32% after the first cycle. After the second cycle, Arm A had a response rate of 33%, Arm B had 30% response rate and Arm C had a 75% response rate. There was an excess of toxicities in Arm B, and no significant SAEs in Arm C. These data were reported at the American Society of Clinical Oncology meeting in June of 2020. Vorinostat has been used extensively in adults and has been granted US FDA approval for the treatment of cutaneous T-cell lymphoma. The approved adult dose is 400 mg orally once daily. Vorinostat is not FDA approved for use in neuroblastoma.
RATIONALE: Low dose deferasirox may be safe and effective in treating patients who have undergone hematopoietic stem cell transplant and have iron overload. PURPOSE: This pilot clinical trial studies safety and tolerability of deferasirox in hematopoietic stem cell transplant recipients who have iron overload. Effect of low dose deferasirox on labile plasma iron is also examined.
To date, no curative option exists for patients with relapsed or refractory stage IV neuroblastoma after previous autologous stem cell transplantation. Our preliminary results of RIC allo-HSCT (protocol RICE) indicate the feasability and low toxicity of allograft in heavily pre-treated children. Furthermore RIC SCT and immunomagnetic CD3/CD19 graft depletion may allow HHCT with lower toxicity and faster engraftment. CD3/CD19 depleted grafts not only contain CD34+ stem cells but also graft-facilitating cells, CD34- progenitors, dendritic and natural killer cells which may allow stable engraftment and participate to GvT effect. After haploidentical stem cell transplantation anti tumour activity exerted by donor derived NK cells could be stimulated by NK cells injections. Those effects may help to reduce the relapse rate and to impove the outcome of those patients. The investigators prospectively evaluated engraftment and immune reconstitution.
This phase II trial is studying the side effects of and how well alisertib works in treating young patients with relapsed or refractory solid tumors or leukemia. Alisertib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Background: - Vorinostat and bortezomib are anti-tumor drugs that have been approved by the Food and Drug Administration to treat different kinds of myeloma and lymphoma in adults. The combination of these two drugs has been tried in a small number of adults, but it has not been formally approved and is experimental, particularly in children. Researchers are interested in determining safe and effective treatment doses of vorinostat and bortezomib in children, and learning more about how these drugs affect tumor growth and human development. Objectives: - To determine safe and effective doses of vorinostat and bortezomib to treat solid tumors in children. - To study the effects of vorinostat and bortezomib on blood cells, blood flow, and human development. Eligibility: - Children, adolescents, and young adults between 1 and 21 years of age who have been diagnosed with solid tumors that have not responded to treatment. Design: - Eligible participants will be screened with a physical examination, blood and tumor samples, and imaging studies. - Participants will have 21-day treatment cycles of vorinostat and bortezomib. Vorinostat will be given as either tablets or liquid doses on days 1 through 5 and 8 through 12 of each cycle. Bortezomib will be given as an intravenous injection on days 1, 4, 8, and 11 of each cycle. Participants will keep a drug administration diary to record information about side effects or other problems with the treatment. - Participants may continue to receive vorinostat and bortezomib for up to 2 years unless serious side effects develop or the tumor does not respond to treatment. - Additional blood samples will be taken at regular intervals for the first 3 days after the first bortezomib dose and for the first 2 days after the first vorinostat dose of the first treatment cycle.
Background: - Pazopanib, a drug that inhibits the growth of new blood vessels in tumors, was recently approved by the Food and Drug Administration to treat advanced kidney cancer in adults. Pazopanib has been used in only a small number of adults, and more research is needed on whether it is safe and effective to use in children. Researchers are interested in determining safe and effective treatment doses of pazopanib in children, and in other studies will examine which form of pazopanib treatment (tablet or liquid) is most effective and well tolerated. Objectives: - To determine a safe and effective dose of pazopanib to treat solid tumors in children. - To study the effects of pazopanib on blood cells, blood flow, and human development. Eligibility: - Children, adolescents, and young adults between 1 and 21 years of age who have been diagnosed with solid tumors that have not responded to treatment. Design: - Eligible participants will be screened with a physical examination, blood and tumor samples, and imaging studies. - Participants will receive pazopanib tablets for 28-day cycles of treatment. Pazopanib should be taken on an empty stomach, at least 1hour before or 2 hours after a meal. Participants may receive pazopanib for up to 24 cycles unless the tumor does not respond or participants develop serious side effects. - Blood samples will be taken on days 1, 15, 22, and 27 of the first cycle of pazopanib, with additional samples taken every 8 weeks during subsequent cycles. - An optional part of the study will collect additional blood samples at regular intervals for 24 hours after the first dose of pazopanib and at regular intervals after another dose during the second or third week of the first treatment cycle.
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.
Neuroblastoma (NB), a common cancer of early childhood originating from primitive sympathetic neural precursors, is characterized by the remarkable heterogeneity of clinical behaviors from spontaneous regression to rapid progression and death. The current therapeutic options are developed according to the Children's Oncology Group (COG) risk stratification criteria based on clinical and biological factors, including tumor stage, MYCN status, age at diagnosis, histology, and ploidy status. 1-2 The treatment strategies ranging from observation alone to intensive multimodality therapy depends on the risk stratification of three subgroups of low, intermediate, and high risk of death. Despite a number of molecular and biologic factors has been identified to predict the prognosis, MYCN amplification, which occurring in roughly 20% of primary NB, is one of the most powerful prognostic factors.3 The co-opting neurotrophin pathways including the neurotrophin receptors (TrkA, TrkB, and TrkC) and their ligands (NGF, BDNF, and neurotrophin-3, respectively), which regulate the differentiation, apoptosis, and growth of neural cells, are also important molecules related to the prognosis of NB.4 However, a proportion of patients with MYCN nonamplified NB still presents clinically aggressive progression similar to those of MYCN amplified tumors, suggesting that other unfavorable molecules exist for the inferior survival.5-6 The IGF-II RNA-binding protein 3 (IMP3), also known as L532S or K homology domain-containing protein overexpressed in cancer (KOC), is a member of RNA-binding protein family which includes IMP1, IMP2, and IMP3. The IMPs are primarily expressed during early embryogenesis and have been implicated in various post-transcriptional functions, including mRNA localization, cell growth, and cell migration during early embryogenesis.7-8 The IMP3 orthologue Vg1-RBP in Xenopus has also been described to promote migration of neural crest cells.9 Recently, the IMP3 is considered an oncofetal protein by increasing proliferation and invasion in various cancers including pancreas, kidney, and lung cancers.10-14 The expression of IMP3 is, however, low or undetectable in adjacent benign tissues.13 These lines of evidence indicate that IMP3 is capable of a potential biomarker to predict cancer progression and metastasis, and may serve as a target molecule for cancer therapy.14 oligonucleotide microarray is a powerful tool to do a genome-wide screening of candidate genes related to cancer prognosis.15 In this study, 22 primary NB tumors were subjected to oligonucleotide microarray analysis. Among the differentially expressed genes according to the patients' prognosis, IMP3 showed an especially high expression level in NB tumors carrying unfavorable prognosis. Further evaluation of IMP3 expression in a large sample size demonstrated that IMP3 expression could predict an unfavorable prognosis of NB patients independent of other biomarkers. Targeting of IMP3 expression in a NB cell line did suppress cell invasion ability, suggesting that IMP3 could not only be a prognostic factor, but also be a potential therapeutic target of NB.
This research trial studies the genes biomarkers in children with neuroblastoma. Studying the genes in a child's cancer cells may help doctors improve ways to diagnose and treat children with neuroblastoma.
The purpose of this study is to find how good and how safe the combination of irinotecan, temozolomide and bevacizumab is for patients with resistant or recurrent neuroblastoma. These drugs have each been given separately to patients, but they have never been given all together. Irinotecan and temozolomide are two drugs that have been used together to treat neuroblastoma in many people. These drugs are considered chemotherapy. Bevacizumab is another drug used to treat cancer. It is made by a company called Genentech. Bevacizumab is an antibody. Antibodies are proteins that are found in the blood and can attach themselves to bacteria and viruses. Bevacizumab attaches itself to a special protein in the bloodstream. This protein helps tumors grow new blood vessels. Blood vessels carry nutrients to feed the tumor. Bevacizumab is thought to block this growth of new blood vessels and starve tumors. It has been used for the treatment of many cancers in adults. It is approved by the FDA for the treatment of adults with colon cancer and other cancers but not for people with neuroblastoma. There is only a small amount of information known on using this drug in children. It has been used with irinotecan before to treat cancer but not in children with neuroblastoma.