Active clinical trials for "Neurofibromatoses"

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining methotrexate with vinblastine may be effective treatment for neurofibromatosis type 1 associated with progressive plexiform neurofibromas. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have neurofibromatosis type 1 associated with progressive plexiform neurofibromas.

The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with idiopathic reading disabilities do, and to determine which intervention is best for particular learner profiles.

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.

RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas. PURPOSE: Phase I trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and plexiform neurofibroma.

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.

Patients with Neurofibromatosis type 2 (NF2) almost always develop acoustic neuromas in both ears. As a result of the tumours themselves or the treatment of the tumours, many patients become profoundly deaf in both ears. It is possible to restore a degree of hearing by placing a hearing implant in the brainstem adjacent to the hearing pathways within the brain. This is termed an auditory brainstem implant (ABI). The aim of this study is to find the optimum head position in relation to the MRI magnet field, which would produce least area of image distortion in patients with ABIs. This will be of benefit in optimising the MR imaging in future patients with implants.

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.

This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects are related to treatment. Plexiform tumors arise from nerves; the only effective treatment is surgical removal. Often, however, not all the tumors can be removed, because of their number or location. Patients with NF1 have a reduced amount of the protein neurofibromin. Neurofibromin is thought to help control the activity of another protein, called ras, which regulates cell growth. Too little neurofibromin, therefore, may allow for uncontrolled cell growth and tumor formation. R115777 interferes with the function of the ras and other proteins. In test tube and animal studies, R115777 has blocked the growth of cancer cells. This study will examine whether the drug is effective against plexiform tumors. Patients with NF1 and progressive plexiform neurofibromas between 3 and 25 years of age may be eligible for this study. Patients whose tumors can be successfully removed surgically may not participate in this study. Candidates are screened with a medical history and physical and eye examinations, blood and urine tests, and magnetic resonance imaging (MRI). Photographs are taken of tumors visible on the body surface. Study participants are randomly assigned to receive either R115777 or placebo (an inactive substance). They take R115777 or placebo tablets every 12 hours for 21 days, followed by a 7-day rest period. This constitutes one 28-day treatment cycle. Treatment continues for as long as the tumors remain stable or shrink and side effects are tolerable. The treatment is switched (for example, from placebo to R115777) or stopped if the tumors grow or if side effects become unacceptable. Patients (or their parents) keep a record of side effects. For the first 3 treatment cycles, patients have a physical examination and blood tests every other week. Blood tests are also done before starting treatment, and at one time point after at least 14 days of treatment to measure the effect of R115777 on proteins in blood cells. A blood sample is obtained before starting treatment and before cycles 4, 7 and 10 and then after every 6 cycles to measure the level of a substance called nerve growth factor. The analysis of nerve growth factor is used to determine if it can predict which patients might be at risk of developing side effects from R115777.

A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.

This study has two primary objectives. The first is to determine if it is feasible and reliable for children and families with a diagnosis of Neurofibromatosis Type 1 (NF1) to use of blood pressure (BP) monitor at home. The second is to determine if there is a difference between a child's measured home BP using standard instructions or using a clinical hypnosis script. This will be determined by a randomised control trial design. Standard and hypnosis Home BP will be compared to the gold standard measurement of BP measured by a trained health care professional in clinic. Children who participate will complete a clinic-based BP with a health care professional, then will be randomised into either the standard home BP measurement or using a hypnosis script prior to BP measurement.