25 Clinical Trials for Various Conditions
In this research study the investigators want to learn more about an alternate, local treatment for skin schwannomas. Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for NF2-related skin schwannomas, ultimately reducing the risks and costs associated with standard surgical removal of such skin tumors if successful.
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas.
This phase II MATCH treatment trial identifies the effects of VS-6063 (defactinib) in patients whose cancer has a genetic change called NF2 mutation. Defactinib may block a protein called FAK, which may be needed for cancer cell growth when NF2 mutations are present. Researchers hope to learn if defactinib will shrink this type of cancer or stop its growth.
This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with associated progressive tumors of vestibular schwannomas (VS), non-vestibular schwannomas (non-VS), meningiomas, and ependymomas. This Master Study is being conducted as a "basket" study that may allow people with multiple tumor types associated with NF2-SWN to receive new drugs throughout this study. Embedded within the Master Study are individual drug substudies. * Investigational Drug Sub-study A: Brigatinib * Investigational Drug Sub-study B: Neratinib
This randomized controlled trial for resiliency training in adolescents with Neurofibromatosis 1 and Neurofibromatosis 2 via secure live video to determine the efficacy two stress and symptom management programs. Both programs are 8 week group programs.
Patients with clinically confirmed neurofibromatosis type 1 (NF1) or neurofibromatosis type 2 (NF2) or a known neurofibromatosis (NF) mutation aged 5 years and above will be eligible to participate and will be recruited from the neurofibromatosis clinic. Given the need for identifying measures that can reliably and sensitively measure focal muscle weakness and allow for measuring muscle strength as a functional outcome in therapeutic clinical trials in NF, this pilot study will assess the reliability of measuring muscle strength in NF1 and NF2 using a hand-held dynamometer.
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
The aim of this study is to determine, whether the 3RP is feasible, accepted and efficacious and durable when delivered via Skype to patients with neurofibromatosis type 2 (NF2) who are deaf or have severe hearing loss using Communication Access Realtime Translation (CART) and/or American Sign Language (ASL). This will be a substudy of the IRB-approved protocol #2013P002605. It is the same study except it is looking at a particular sub-population: patients with NF2 who are hard of hearing.
The Nucleus 24 Auditory Brainstem Implant (ABI) is the only FDA approved device for restoration of meaningful hearing in Neurofibromatosis Type 2 (NF2) patients. This device has been discontinued, meaning that there is no commercially approved device currently available. The replacement model, the ABI541 (an unapproved device), is being investigated in ongoing clinical trials. A compassionate use arm of a clinical trial allows patients with NF2 to be implanted with this new ABI.
The purpose of this research study is to look at participants with solid tumor malignancies and specific mutations respond to treatment with everolimus.
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients. RAD001 is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors.
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.
For patients with malignant pleural mesothelioma that has grown despite treatment with standard chemotherapy, no treatment has yet proven beneficial. The purpose of this study is to find out what effects, both good and bad, that everolimus has on the cancer. Everolimus works by blocking a protein that helps the cancer grow. The goal of this clinical research study is to learn if the study drug everolimus can shrink or slow the growth of mesothelioma. The safety of this drug will also be studied. The patients' physical state, changes in the size of the tumor, and laboratory findings taken during the study will help us decide if everolimus is safe and effective.
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system. It causes non-cancerous tumors (which are known as neuromas) to grow on the nerves around a person's body. Some signs of NF2 include a gradual loss of hearing and tumors growing on the skin, the brain and the spinal cord which can lead to complications. Lapatinib is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors. The investigators know a lot about how well it is tolerated, but the investigators do not know if it is effective in treating your condition, therefore it is considered to be an investigational medication. This study will test whether Lapatinib may shrink tumors commonly found in patients with NF2 or stop them from growing. This will help us to decide if Lapatinib should be used to treat NF2 patients in future. Lapatinib is a drug that has been used for over 10 years to treat various forms of cancer. It has not been studied for the treatment of tumors in NF2 patients.
Adults treated with X-rays in childhood for benign conditions such as enlarged tonsils and adenoids have an increased risk of developing nervous system tumors. The risk is highest for schwannomas (RR of 33.1, 95% CI of 9.4-116.5), intermediate for meningiomas (RR of 9.5, 95% CI of 3.5-25.7) and lowest for gliomas (RR of 2.6, 95% CI of 0.8-8.6). Studies of sporadic tumors of these types have demonstrated the presence of somatic mutations in the NF2 gene. Because these mutations are not usually seen in other types of tumors, they are believed to be what caused the sporadic neural tumors to develop. Germline mutations in the same gene are responsible for the autosomal dominant disorder known as neurofibromatosis 2 (NF2) which is characterized by the development of similar types of neural tumors. The purpose of the proposed study is to determine if neural tumors that developed in people who were treated with X-rays in childhood also have somatic NF2 mutations. This will be done using DNA from paraffin-embedded neural tumors that developed in \~ 112 individuals treated with X-rays in childhood for benign head and neck conditions. These individuals are from a cohort of over 4,000 irradiated persons followed by Michael Reese Hospital in Chicago since 1974. If we find NF2 mutations in the radiation-related tumors, we will determine whether they are somatic or germline by looking for NF2 mutations in DNA from buccal cells of the patients with the studied tumors. We expect that most patients will have NF2 mutations only in tumor DNA. However, there is a remote possibility that one or more patients may have a germline NF2 mutation and thus an increased risk of developing neural tumors even in the absence of X-ray treatment. We will then compare the types and frequencies of the somatic NF2 mutations with those found in sporadic neural tumors. If we do not find somatic NF2 mutations in the radiation-related tumors, we will conclude that X-rays caused neural tumors to develop through interactions with another gene or genes. Either result will contribute to our knowledge of radiation tumorigenesis. We will send letters describing this study to patients from the Michael Reese Hospital cohort who have developed radiation-related neural tumors. Those who consent to take part in it will be asked for permission to obtain paraffin blocks from any neural tumor that they have had removed and to donate buccal cells for NF2 mutation studies. Finally, they will also be asked to complete a questionnaire that will help us update their medical history and obtain a medical history on close blood relatives.
OBJECTIVES: I. Define the growth rates and clinical course of NF2-related tumors in patients with neurofibromatosis type 2. Associate growth rate with physical function.
Tumors can grow on the auditory nerves and can cause hearing loss. A common type of tumor that does this is a vestibular schwannoma (VS), or acoustic neuroma. These tumors are not cancerous. Most often, people have only one VS. Occasionally, people have more than one VS and may have a condition called neurofibromatosis type 2 (NF2). Because VS can cause hearing loss, many people with VS will have treatment to preserve their hearing. This treatment usually involves surgery or radiation therapy. There are risks to these procedures, and sometimes they do not work to prevent hearing loss. Because surgery and radiation have risks and are not able to help everyone with VS, 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 VS from growing larger and causing hearing loss, and might possibly even cause the VS to shrink in size. This study is exploring whether a drug that is approved by the FDA and is currently used to treat breast cancer might also work to treat VS. This study will measure the amount of drug that travels from the bloodstream and arrives at the tumor. This drug is safe and has few side effects. If this drug is shown to reach the tumor, it might be used in the future to treat VS without needing surgery or radiation. This study is recruiting people who are having surgery for VS. If you are going to have surgery to treat a VS, you may be eligible to participate.
This is a Phase 1, first-in-human (FIH) clinical study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of IK-930, an oral TEAD inhibitor, administered orally (PO) as monotherapy in subjects with advanced solid tumors with or without gene alterations in the Hippo pathway for whom there are no further treatment options known to confer clinical benefit. The study consists of two phases, an initial Dose Escalation phase followed by a Dose Expansion phase.
This phase II trial studies how well vismodegib, focal adhesion kinase (FAK) inhibitor GSK2256098, and capivasertib work in treating patients with meningioma that is growing, spreading, or getting worse (progressive). Vismodegib, FAK inhibitor GSK2256098, capivasertib, and abemaciclib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection. AR-42 is a small molecule which crosses the blood brain barrier (BBB) in rodents, but the investigators are not certain yet if it will penetrate human VS. Meningiomas are outside the BBB, but seem to be unusually resistant to all current medical treatments. The primary endpoint of the bioactivity of suppression of p-AKT by AR-42 was selected as drug activity seems more informative than bioavailability. Our preclinical data and others have shown dose dependent suppression of p-AKT by AR-42 in both VS and meningiomas.
The NF Registry is a database of patient-reported symptoms, treatments, and experiences with their neurofibromatosis disease. It is a contact registry to relay clinical trial opportunities to targeted patient subgroups, and to supply de-identified disease data to researchers. It has the potential to become a natural history resource.
Background: * Neurofibromatosis type II (NF2) is associated with tumors of the nerves, brain, and spinal cord. Most people with NF2 develop vestibular schwannomas, or tumors on the hearing and balance nerves. As they grow, vestibular schwannomas can cause hearing loss and balance problems. If they grow very large they can cause more serious problems, such as seizures, loss of eyesight, weakness, speech problems, and problems with the sense of touch. More research is needed into NF2 because researchers do not completely understand why these tumors occur or what makes them grow over time. * Currently, tumor size is measured with magnetic resonance imaging (MRI) scans. However, MRI scans cannot predict how fast a tumor will grow. By using positron emission tomography (PET) scanning, researchers hope to be able to predict sudden growth spurts of tumors associated with NF2 and develop better treatment methods for this type of cancer. Objectives: - To use magnetic resonance imaging and positron emission tomography to better understand the growth of brain tumors in people with neurofibromatosis type II. Eligibility: - Individuals between 18 and 50 years of age who have been diagnosed with NF2 and have at least three untreated intracranial tumors. Design: * This study requires an initial set of outpatient visits to the NIH Clinical Center that will last 7 to 10 days. * Participants will have a physical and neurological examination and blood tests at the first visit. Participants will then have the following imaging studies to examine the tumors: * MRI scans of the brain * PET scans of the brain, combined with a computed tomography (CT) scan. The PET scans will be performed on separate days. Different contrast agents will be used for both scans, so researchers will inform participants if they need to fast or follow other procedures before having the scan. * After the initial imaging studies, participants will have additional MRI scans every 6 months for 2 years to track tumor growth.
The purpose of this study is to characterize the safety and tolerability of IAG933 in patients with mesothelioma, NF2/LATS1/LATS2 mutated tumors and tumors with functional YAP/TAZ fusions and to identify the maximum tolerated dose and/or recommended dose.
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in children who are deaf and cannot receive a cochlear implants.
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in persons who are deaf in both ears and are not candidates for cochlear implants.