Active clinical trials for "Neurofibromatoses"

To demonstrate the initial feasibility and determine preliminary impact on clinical outcomes of the iCanCope-NF program in a pilot RCT. If successful this pilot study will support conducting a larger randomized control trial (RCT). The primary research question is what is the feasibility of the iCanCope-NF program? The investigators define feasibility as (1) rates of accrual and dropout, daily log-ins, engagement, and outcome measures completed and (2) perceptions regarding intervention acceptability and satisfaction; and what are the levels of engagement. log-ins, with the intervention? The secondary questions are: (1) how does the iCanCope-NF program compare with the control condition in differences of pain and pain-related activity limitations, sleep functioning, emotional functioning (depression, anxiety), opioid usage, pain catastrophizing, self-efficacy, respondent burden (i.e. Physical Functioning, R, Vitality, Social Functioning, Role-Emotional, and Mental Health), and psychological flexibility immediately post-treatment (T2), (2) does the iCanCope-NF + CM increase the engagement of the iCanCope-NF program as compared to iCanCope-NF without CM, and do their corresponding levels of pain and pain-related activity decrease with CM?, and (3) do individuals with NF1 utilize the MBAA to help reduce pain symptoms? The investigators hypothesize that by customizing and including MBAA to the program for adults with NF1, that individuals who engage regularly as seen through Analytics Platform for Evaluating Effective Engagement (APEEE) application, will acquire new sets of skills to facilitate pain management, while pain as reported with the Brief Pain Inventory will decrease.

This is a phase I open label study designed to evaluate the safety, tolerability, PK and efficacy of selumetinib in Japanese paediatric patients with neurofibromatosis type 1 and inoperable and symptomatic plexiform neurofibroma.

Background: Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause frequent and significant pain. This pain can have a severe impact on a person s quality of life. Acceptance and Commitment Therapy (ACT) is a new type of therapy that may help people manage pain. It teaches behavioral techniques that can be used to change how people react to pain. ACT may help people with NF1 cope better with the pain associated with the disorder. Researchers want to test ACT techniques to see if they can improve coping in people with NF1 pain. Objectives: - To see if ACT can help treat chronic pain in adolescents and young adults who have NF1. Eligibility: - Adolescents and young adults between 12 and 21 years of age who have chronic pain associated with NF1. Design: Participants will be screened with a physical exam and medical history. They will also complete a set of questionnaires about their pain levels, quality of life, and medications that they are taking. Participants will take part in a training workshop for ACT. It will consist of three 2-hour sessions over 2 consecutive days. The workshop will teach techniques for setting goals that reflect personal values, and changing the focus from pain avoidance to pain management. There will be practice exercises between each session. At the end of the workshop, participants will receive a workbook with the exercises to continue to practice at home. Participants will be encouraged to spend at least 20 minutes, three times per week, on these practice exercises. There will be a followup phone call to check on pain management after the workshop. Three months after the first study visit, participants will complete the study questionnaires again from home. They will provide more information about how they are managing their pain. The questionnaires will be returned by mail in an envelope provided by the study team.

GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin. SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and 633 nm light in the treatment of benign dermal neurofibromas. Specifically, the primary goal of the current study is to determine the maximum tolerable light doses that can be administered to subjects undergoing topical photoillumination photodynamic therapy with standard application of Levulan Kerastick (ALA) for Topical Solution.

People who have neurofibromatosis type 2 (NF2) can have tumors that grow on the auditory nerves and cause hearing loss. These tumors are called vestibular schwannomas (VSs), or acoustic neuromas. People with NF2 can also get schwannomas in other parts of their body, as well as tumors called meningiomas and ependymomas. Because VSs can cause hearing loss, many people with NF2 will have treatment to preserve their hearing. This treatment usually involves surgery. Because surgery has risks and is not able to help everyone with VSs, other methods of treatment are being explored. One area of exploration is looking to see if there is a drug that can be taken that might prevent the VSs from growing larger and causing hearing loss or brainstem compression. This study is exploring whether a drug that is approved by the FDA and is currently used to treat other tumors might also work to treat VSs. Based on people who have taken this drug to treat VSs already, there is some reason to think that it might be helpful to certain people with NF2. People enrolled in this study will receive the drug one time every three weeks for one year by infusion. This study will follow subjects over the course of the year that the person is taking the drug and for six months after the drug is stopped. This study is recruiting people who have NF2 and are currently having symptoms of tinnitus, dizziness, and/or hearing loss from their VSs. If you have NF2 and are currently having symptoms caused by your VSs, you may be eligible to participate.

This study will evaluate the feasibility of combining two drugs, Tarceva (an anti-EGFR agent), and Rapamycin (an mTOR inhibitor), in children with progressive low-grade gliomas who have failed initial conventional treatment. In addition to evaluating the toxicity of this drug regimen, the potential efficacy of the regimen will be assessed.

The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. It targets a substance in the body called VEGF (Vascular Endothelial Growth Factor). VEGF helps tumors to grow and survive by supporting the growth of blood vessels that bring nutrients to the tumor. VEGF is made by cancerous tumors and also by non-cancerous tumors such as neurofibromas.

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.

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: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a patient's response to treatment, and help plan the most effective treatment. PURPOSE: This diagnostic trial is studying how well FDG-PET and MR perfusion imaging work in finding disease progression and determining response to treatment in patients with neurofibromatosis 1 and plexiform neurofibroma.