View clinical trials related to Neurofibroma.
Filter by:This study is for slow growing tumors called plexiform neurofibromas (PNF) which are a relatively common problem in people with neurofibromatosis type 1 (NF1). These tumors are benign but as they grow, they can become disfiguring as well as disabling or even life threatening. They often cause pain, difficulty using arms or legs because of spinal cord compression, and/or nerve damage. At present, the only available therapy for plexiform neurofibromas is to try to surgically remove as much of the tumor as is possible. Because these tumors grow into the surrounding areas, total surgical resection is often impossible. Most tumors will re-grow after surgery if the entire tumor cannot be removed. To date, other treatments including chemotherapy and radiotherapy have not been able to shrink these tumors. Interferon is a drug that is used for different types of tumors as well as for hepatitis. It has been used in the treatment of plexiform neurofibromas (PNF) with some subjects showing improvement in symptoms and/or a decrease in the size of the tumor. Most subjects had no further growth of their tumor while on the PEG-Intron. The drug used in this study is PEG (pegylated)-Intron. PEG-Intron is a long acting form of interferon which keeps the drug from being broken down in the body for a longer period of time and potentially could be more effective than the short-acting interferon. PEG-Intron has been approved by the Food and Drug Administration (FDA) for the treatment of Hepatitis C. The goals of this study are: 1. To determine how your child's plexiform neurofibroma responds to PEG-Intron when given weekly. 2. To determine the side effects of PEG-Intron when given weekly to participants with plexiform neurofibromas. 3. To evaluate a new method of measuring changes in the size of tumors called volume analysis. This method measures the entire volume of a tumor in three dimensions. The standard method of measuring tumors uses only the length and/or width of the tumor. By studying the different ways of measuring tumors the investigators hope to be able to determine which method is the most accurate and useful.
Neurofibromatosis type I (NF1) is a genetic disorder that affects approximately 1 in 3500 individuals. Half of people with NF1 inherit the condition from a parent, and half have a new occurrence of the condition. The manifestation of NF1 is highly variable and multiple organ systems are typically affected. Some of the more common symptoms include benign neurofibromas, café au lait spots, Lisch nodules (tan spots on the iris of the eye). Some individuals with NF1 also exhibit more severe associated conditions, such as optic pathway tumors (gliomas) or bones bending or curving. Neurocognitive deficits and specific learning disabilities occur in approximately 30 to 50% of individuals with NF1 and are regarded by some observers and sufferers to be among the most troubling features of a disease. The most commonly reported findings are deficits in visuoperceptual ability, motor coordination, expressive and receptive language, and executive functioning, which requires intact short-term memory and attention. Patients with NF1 also show a slight depression in mean IQ scores compared to healthy adults without the disorder. While cognitive deficits are now a widely-recognized feature of Neurofibromatosis Type 1 (NF1), the precise cause of these deficits still remain to be determined. Dr. Alcino Silva, a co- investigator on this study, has developed an animal model of NF1 in which mice have a specific mutation of the *NF1* gene. These mice are physically normal but show specific learning impairments. Dr. Silva's lab found that treatment with a medication called lovastatin, a drug typically used for high cholesterol, reversed some of the spatial deficits seen in these animals. Lovastatin is a medication commonly used to treat high cholesterol and has been proven to be relatively safe and tolerable in humans. The investigators are now conducting a randomized, double-blinded, placebo- controlled, trial of lovastatin in patients with NF1. Participants will be randomly assigned to lovastatin or placebo and treated for approximately 14 weeks with baseline and follow-up assessments to evaluate safety and any effects on neurocognitive test performance.
RATIONALE: Drugs used in chemotherapy, such as vinblastine and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. PURPOSE: This phase I trial is studying the side effects and best dose of vinblastine when given together with carboplatin in treating young patients with newly diagnosed or recurrent low-grade glioma.
This study will construct tissue microarrays (TMAs) pertaining to childhood cancer. TMA technology is a recently developed one that allows for evaluating hundreds of tissue samples simultaneously on the DNA, RNA, and protein levels. The goal is to identify a potential molecular signature. Cancer drug discovery is currently focused on identifying drugs targeted at the molecular level. Such drugs would be more selective and specific for proteins and signaling pathways that are directly involved in the origin of tumors. However, the origin of cancer among adults differs from that of cancer diagnosed in children. The overall approach by the pharmaceutical industry in developing drugs is not likely to be aimed at low-incidence cancers, such as childhood cancers. Thus, the researchers in this study propose to create a childhood cancer TMA that include specimens from a wide range of solid tumors that present a poor prognosis for patients. This TMA would in turn be used to identify antibodies and lead to developing molecularly targeted drugs that would reach clinical trials in adults. TMAs are created robotically. Small tissue cores are taken from paraffin-embedded tissue blocks and are implanted into new paraffin blocks. The recipient blocks are then processed to produce several hundred specimens that can be evaluated on a single glass slide. Specimens representing 17 distinct kinds of pediatric solid tumors will be used in this study. Also included will be samples of plexiform neurofibroma-that is, benign growths of nervous and connective tissues. Tissue specimens will come from patients who were age 25 or younger at the time of diagnosis of their cancer or plexiform neurofibroma. No procedures will be performed for the sole purpose of obtaining tissue for this study. The TMA developed in this study will not be commercialized. The results for individuals whose tumor specimens are used in the array will not be sent to patients or their treating physicians.
This phase II trial is studying how well AZD2171 works in treating patients with neurofibromatosis type 1 and plexiform neurofibroma and/or neurofibroma near the spine. AZD2171 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.
This study will explore the growth of dermal neurofibromas (skin tumors) in patients with neurofibromatosis type 1 (NF1). Investigators will try to learn: 1) how fast (or slow) these benign tumors grow in NF1, 2) how often new tumors appear and 3) what genes are involved in the growth of the tumors. Men and women between 20 and 50 years of age diagnosed with NF1 and their biological parents are eligible for this study. Patients with NF1 are evaluated at the NIH Clinical Center with the following tests and procedures: - Medical examination and drawing of family tree. - Photos of the back, abdomen and thigh in order to count the number of skin tumors. - Photos of the skin taken with a special camera (Primos camera) that takes very detailed pictures of a small area of skin. - Photos of the skin taken with a dermatoscope, which takes very detailed pictures of a small area of skin under high magnification. - Biopsy of at least one skin tumor and biopsy of a small piece of normal skin. - Blood sample collection for genetic testing of the gene NF1 and to establish a cell line. - Other medical tests (e.g., x-rays or MRI) if needed. Patients and their families will also have a genetic counseling session and an opportunity to ask questions about neurofibromatosis type 1. Patients return to the NIH after 3, 6, 12, 18 and 24 months for follow-up photographs and possibly blood samples. Biological parents of patients provide a blood sample for genetic testing.
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving combination chemotherapy with or without radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving combination chemotherapy after surgery may kill any tumor cells that remain after surgery. PURPOSE: This phase II trial is studying how well combination chemotherapy works in treating patients with stage III or stage IV malignant peripheral nerve sheath tumors.
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1. We will obtain information on the natural history, burden, functional and health status, health-related quality of life, and surgical interventions/outcomes of tibial dysplasia. The project will also establish a Core Facility (NOCF) for tissue samples for future studies.
RATIONALE: PEG-interferon alfa-2b may interfere with the growth of tumor cells. PURPOSE: This phase I trial is studying the side effects and best dose of PEG-interferon alfa-2b in treating young patients with plexiform neurofibroma.
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. We, the researchers at Hospices Civils de Lyon, designed a randomized, double blind, placebo controlled, crossover trial with a total follow-up duration of 9 weeks to evaluate the effect of methylphenidate (MPH) on the improvement on the simplified parents Conners' Rating Scale. In a parallel exploratory study we will compare the nature of attention deficit disorders in NF1 children to 30 ADHD NF1-free controls. Children aged 7 to 12 years are eligible when their intelligence quotient (IQ) is between 80 and 120. Fifty subjects (25 for each period) were required for testing the primary study hypothesis.