DICER1-related Pleuropulmonary Blastoma Cancer Predisposition Syndrome: A Natural History Study...
Pleuropulmonary BlastomaCystic Nephroma3 moreBackground: - Pleuropulmonary blastoma (PPB) is a rare fast-growing lung tumor that is associated with other, rare tumor types. Most cases of PPB appear in children younger than 6 years of age. Recently, it has been shown that this condition can be inherited (e.g., mutation of the DICER1 gene). Researchers are studying both clinical and genetic aspects of this newly described condition. They are interested in collecting further medical history and genetic information on individuals and close relatives of individuals who have PPB or other rare associated tumors. Objectives: - To study individuals with a personal or a family history of pleuropulmonary blastoma (PPB) or other rare tumors that can be associated with PPB (e.g., cystic nephroma, nasal chondromesenchymal hamartoma, ovarian Sertoli-Leydig cell tumors, ocular medulloepithelioma). Eligibility: Individuals who have been diagnosed with PPB and/or PPB-related tumors. Close blood relatives (e.g., parents, siblings, grandparents) of individuals who have been diagnosed with PPB and/or PPB-related tumors. Design: Interested participants can enroll or inquire about this study by calling 1-800-518-8474. Participants will be asked to complete family history and medical history questionnaires. They will complete the questionnaire if they are at least 18 years of age, or another person will complete the questionnaire if the key family member is too young to do so on his or her own. Participants will be asked to sign a medical record release form to allow researchers to examine detailed medical history information. Participants may be asked to have a physical examination and imaging studies, provide blood and saliva samples, or provide tumor tissue from prior biopsies or cancer surgeries. Annually, participants will update the family history and individual information questionnaires to document important changes in medical history, and will also update the medical record release form. Participants may be asked to provide additional cheek lining cells and/or blood samples, as well as tumor tissue from any new or planned biopsies or tumor surgeries. Treatment will not be provided as part of this protocol....
Phase I/II: Decitabine/Vaccine Therapy in Relapsed/Refractory Pediatric High Grade Gliomas/Medulloblastomas/CNS...
GliomasMedulloblastoma2 moreThe main purpose of this study is to determine the safety of using the combination of decitabine and a cancer vaccine plus Hiltonol. The vaccine will be made from the subject's blood cells and is designed to interact in the subject's body with cells that are programmed to fight specific tumor proteins NY-ESO-1, Melanoma Antigen Gene-A1 (MAGE-A1) and Melanoma Antigen Gene-A3 (MAGE-A3). The decitabine will be given to increase the amount and activity of these cancer proteins on the surface of tumor cells to increase the possibility that the vaccine will stimulate cells to act against the tumor cells. Subjects will be assessed to determine how these tumors respond to the treatment.
Gamma-Secretase Inhibitor RO4929097 in Treating Young Patients With Relapsed or Refractory Solid...
Childhood Atypical Teratoid/Rhabdoid TumorChildhood Central Nervous System Choriocarcinoma45 moreThis phase I/II clinical trial is studying the side effects and best dose of gamma-secretase inhibitor RO4929097 and to see how well it works in treating young patients with relapsed or refractory solid tumors, CNS tumors, lymphoma, or T-cell leukemia. Gamma-secretase inhibitor RO4929097 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Palbociclib Isethionate in Treating Younger Patients With Recurrent, Progressive, or Refractory...
Childhood Choroid Plexus TumorChildhood Ependymoblastoma17 moreThis phase I trial studies the side effects and best dose of palbociclib isethionate in treating younger patients with central nervous system tumors that have grown, come back, or not responded to treatment. Palbociclib isethionate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
A Study to Determine the Activity of Robatumumab (SCH 717454) in Participants With Relapsed Osteosarcoma...
OsteosarcomaSarcoma2 moreParticipants with relapsed osteosarcoma that can be treated with surgery will be randomized to robatumumab administered intravenously (IV) at one of two dose levels. These participants will first receive robatumumab, have surgery performed, and continue to receive treatment every two weeks until a year of dosing, or until disease progression. Participants with unresectable osteosarcoma or Ewing Sarcoma will receive robatumumab IV once every two weeks until disease progression. Participants who achieve a complete response (CR) or partial response (PR) after tumor evaluations may undergo surgical resection. After surgery, participants are eligible to receive 10 mg/kg robatumumab until disease recurrence/progression or one year of total dosing, whichever occurs first.
Antineoplaston Therapy in Treating Patients With Metastatic, Recurrent, or Refractory Primitive...
Primitive Neuroectodermal TumorCurrent therapies for Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors.
Chemotherapy in Treating Patients With Metastatic Ewing's Sarcoma or Primitive Neuroectodermal Tumor...
SarcomaRATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have metastatic Ewing's sarcoma or primitive neuroectodermal tumor.
Combination Adenovirus + Pembrolizumab to Trigger Immune Virus Effects
Brain CancerBrain Neoplasm11 moreGlioblastoma (GBM) and gliosarcoma (GS) are the most common and aggressive forms of malignant brain tumor in adults and can be resistant to conventional therapies. The purpose of this Phase II study is to evaluate how well a recurrent glioblastoma or gliosarcoma tumor responds to one injection of DNX-2401, a genetically modified oncolytic adenovirus, when delivered directly into the tumor followed by the administration of intravenous pembrolizumab (an immune checkpoint inhibitor) given every 3 weeks for up to 2 years or until disease progression. Funding Source-FDA OOPD
NAC to Prevent Cisplatin-induced Hearing Loss
Neuroectodermal TumorsPrimitive3 moreCisplatin is a key chemotherapy agent for the treatment of multiple childhood cancers but causes permanent hearing loss. This study investigates the drug N-acetylcysteine (NAC) to determine the dose necessary to protect hearing and also how well tolerated NAC is when combined with chemotherapy.
Vorinostat and Temozolomide in Treating Young Patients With Relapsed or Refractory Primary Brain...
Childhood Atypical Teratoid/Rhabdoid TumorChildhood Central Nervous System Choriocarcinoma32 moreThis phase I trial is studying the side effects and best dose of vorinostat when given together with temozolomide in treating young patients with relapsed or refractory primary brain tumors or spinal cord tumors. Vorinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Vorinostat may help temozolomide work better by making tumor cells more sensitive to the drug.