View clinical trials related to Neurofibroma.
Filter by:This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative NF1-associated PNs.
The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD
Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9 technology. 3. Differentiate and characterize disease-relevant brain cells such as excitatory and inhibitory neurons, astrocytes and oligodendrocytes from patient-specific iPSC lines. 4. Screen and identify the drug(s) that can reverse or alleviate the disease phenotypes.
Background: Gastrointestinal stromal tumors (GIST) can cause serious medical problems. The only known treatment is surgery. But completely removing a GIST tumor with surgery is often not possible. Researchers want to see if a new drug, selumetinib, can help treat these tumors. Objective: To find out if selumetinib shrinks or slows the growth of GIST tumors and to see its side effects. Eligibility: People ages 3 and over who have one or more GIST tumors and may have neurofibromatosis type I (also called NF1). Their NF1 GIST has shown some growth or cannot be completely removed with surgery. Design: Participants will be screened with heart and eye tests and scans. Participants will be told what foods and medicines they cannot take during the study. Participants will keep a diary of the medicine they take during the study. Participants will take selumetinib capsules twice daily on an empty stomach for 28 days in a row. This is 1 cycle. During the cycles, participants will have study visits. These may include: Medical history Physical exam Blood and urine tests Heart tests Scans of their tumors Eye exam Positron emission tomography scan. They will be get radioactive glucose an IV line. They will lie quietly in a darkened room for 50-60 minutes then have the scan. Participants will answer questions about how they are feeling. Participants can stay in the study until they have bad side effects or their tumor grows. After finishing treatment, participants will be watched for side effects for 30 days.
Background: Saliva, blood, tissue, and cancer contain DNA. DNA makes the "instruction book" for the cells in the body. Cancer is caused by changes in DNA that affect cell function. Researchers want to test DNA of people with tumors. They want to look for genetic changes in tumors that could be targets for treatment. Because DNA can change as cancer changes, more testing may be done at different times. Objectives: To find the DNA changes in cancer that may help guide treatment. To collect samples and data to be used in future studies. Eligibility: People any age with cancer or a pre-cancerous tumor Design: - Participants will be screened with a medical history, physical exam, and blood tests. Participants will give a sample of their tumor. This is usually from a previous procedure. Participants will give a saliva or blood sample. They cannot eat, drink, smoke, or chew gum for 30 minutes before giving saliva. They will spit about 1 teaspoon of saliva into a tube. - Some participants may have a punch biopsy instead. A small instrument will take a small piece of skin. - Researchers will collect data from participants medical records. - Participants will answer questions about their family health history. They will also answer questions about their views on the study, including possible unexpected results. - Extra blood or tissue samples may be taken at other times during the participants' treatment. All samples will be saved in secure ClinOmics freezers to be used in future studies. - Participants will be told by their doctors if any test results affect their health or their cancer treatment.
The purpose of this study is to determine if Proton Therapy can provide effective and safe treatment for Malignant Peripheral Nerve Sheath Tumors of the spine and Neurofibromas of the spine.