NF2 Clinical Trials & Studies


  • Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients
    ClinicalTrials.gov Identifier: NCT01490476
    The purpose of the study (in France) is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients.

  • Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2
    ClinicalTrials.gov Identifier: NCT01419639
    This trial studies whether Everolimus is efficacious in treating neurofibromatosis 2.

  • Sorafenib in a NF2 study, in the UK
    Adults who have Neurofibromatosis 2 (NF2) and skin tumours (schwannomas) are invited to participate in a research study being undertaken in Plymouth and Manchester; Professor C. Oliver Hanemann, a consultant neurologist at the Peninsula College of Medicine and Dentistry in Plymouth is the Chief Investigator.....

  • Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
    ClinicalTrials.gov Identifier: NCT01345136.
    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.

  • Using Positron Emission Tomography (PET) to Predict Intracranial Tumor Growth in Neurofibromatosis Type II (NF2) Patients
    ClinicalTrials.gov Identifier: NCT01222728.
    Objective: To use magnetic resonance imaging and positron emission tomography to better understand the growth of brain tumors in people with neurofibromatosis type II.

  • A Phase II Study of Nilotinib in Growing Vestibular Schwannomas
    ClinicalTrials.gov Identifier: NCT01201538.
    Acoustic Neuromas (otherwise known as Vestibular Schwannoma -VS) are benign tumors which grow on the hearing nerve and can cause progressive hearing loss and compression of vital brain structures and even death if it continues. The primary objective of this study is to evaluate the efficacy of Nilotinib in the treatment of patients with progressing VS. Secondary objectives of this study is to evaluate the toxicity profile, quality of life and symptom management of Nilotinib in the treatment of patients with progressing VS.

  • Phase 2 Study of Bevacizumab in Children and Adults With Neurofibromatosis Type 2 and Symptomatic Vestibular Schwannoma
    ClinicalTrials.gov Identifier: NCT01207687.
    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.

  • Phase II Trial of Bevacizumab in Patients With Recurrent or Progressive Meningiomas
    ClinicalTrials.gov Identifier: NCT01125046.
    PURPOSE: This phase II trial is studying how well bevacizumab works in treating patients with recurrent or progression meningiomas.

  • Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
    ClinicalTrials.gov Identifier: NCT00973739.
    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.

  • PTC299 for Treatment of Neurofibromatosis Type 2
    ClinicalTrials.gov Identifier: NCT00911248.
    Formation of new blood vessels (angiogenesis) is important for tumor growth in neurofibromatosis type 2 (NF2). It is known that tumors make a protein called vascular endothelial growth factor (VEGF) and there are higher levels of VEGF in the tumors and blood of many patients with NF2. VEGF stimulates the formation of blood vessels that supply the tumor with nutrients and oxygen. PTC299 is an oral drug that has been shown to decrease production of VEGF in animal models of human cancer. In these animal models, oral PTC299 administration decreases VEGF levels in the tumor and in the bloodstream, decreases blood vessel numbers in the tumor, and significantly slows or halts tumor growth. Safety studies in research animals indicate good tolerability at doses and drug levels that are higher than those planned for the clinical studies. Results from Phase 1a studies in healthy volunteers indicate that PTC299 achieves levels of PTC299 in the bloodstream that are known to be active in animal models of human tumor. This Phase 2 study is designed to test the hypothesis that PTC299 will be tolerable and will show evidence of VEGF reduction, antitumor activity, and hearing improvement when administered orally to patients with NF2
    - PTC's Neurofibromatosis Type 2 Clinical Trial Overview
    - PTC's NF2 Trial FAQs
    - Additional infomation on the PTC299 trial for NF2.

  • Concentration and Activity of Lapatinib in Vestibular Schwannomas
    ClinicalTrials.gov Identifier: NCT00863122.
    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.

  • Natural History Study of Patients With Neurofibromatosis Type 2
    ClinicalTrials.gov Identifier: NCT00598351.
    This study will examine over the long-term the progress of patients with neurofibromatosis Type 2 (NF2), a condition associated with tumors of the nerves, brain and spinal cord. It will study patients' tumors to learn how fast they can grow and if certain factors might affect their growth. It will also examine the effects of the tumors on patients' abilities to carry out activities of daily living.
    People between 8 and 75 years of age with NF2 may be eligible for this study.

  • Neurofibromatosis Type 2 Associated Color Vision Anomalies and Birth Defects: Incidence and Insights
    PURPOSE: To determine the frequency of birth defects and miscarriages in patients with NF2, and to determine the frequency of color blindness in NF2 patients.

  • NF2 Patients Needed for New Vestibular Study at Harvard Medical School / Massachusetts Eye and Ear Infirmary.
    Adrian Priesol, M.D.
    phone: (617) 573-4148
    email: adrian_priesol@meei.harvard.edu
    "The general clinical goal for our study is to quantify motion perception for a range of vestibular disorders. A long-term goal is to develop new clinical tests to provide early diagnosis for vestibular disorders. These clinical research efforts are part of a broader basic science investigation of vestibular thresholds. At this time, we are seeking NF2 patients who have complete loss of vestibular (i.e., “balance/equilibrium organ”) function in both ears following surgical removal of tumors from both ears. Travel reimbursement would be paid for all qualifying volunteers."

Additionally, for a comprehensive listing of US, International, and/or privately supported clinical trials, please see these websites below:

NOTE: try inputing "neurofibromatosis 2".... or a NF2 tumor tissue type such as "Vestibular Schwannoma", "Schwannoma", "Meningioma", "Glioma", "Ependymoma", or "Astrocytoma", for example, within its respective search field.


  • Clinicaltrial.gov - studies and trials with search of "NF2". A service of the U.S. National Institutes of Health.

  • CERN Foundation - Collaborative Ependymoma Research Network (CERN). Clinical trials are an integral part of the CERN Foundation’s many projects and collaboration. Our hope in these trials, and in all of our work, is to collect critical data that will advance a cure.

  • Children's Tumor Foundation (CTF)
    • NF2 trials and studies
    • Clinical trials chart

  • National Institute of Health (NIH) - Search the collection of research studies being conducted at the NIH Clinical Center. Developed by the National Library of Medicine.

  • National Cancer Institute (NCI) - Search NCI's list of 8,000+ clinical trials now accepting participants.

  • International Federation of Pharmaceutical Manufacturers & Associates (IFPMA) - IFPMA Clinical Trials Portal. IFPMA Clinical Trials Portal is brought to you by IFPMA on behalf of its Member Companies and Associations. FPMA Clinical Trials Portal ensures a free and easy-to-use interface for patients and health professionals alike to ongoing clinical trials, clinical trial results and complementary information on related issues.

  • Clinical Trials Search - Find clinical trials by condition, sponsoring organization, or location. Detailed nationwide listings with recruitment status, contact information available free of charge.

  • CenterWatch - CenterWatch lists more than 80,000 active clinical trials seeking study volunteers each month, making it the largest online and dynamic database of global clinical trials involving new drugs and devices regulated by the FDA and the Department of Health and Human Services.

  • Virtual Trials - A premier source of information for brain tumors. As well as hosting many online support groups, chat rooms, newsletters, articles, and a database of clinical trials. Run by the Musella Foundation.

DISCLAIMER - please read:
We are not medical experts nor affiliated with any healthcare organization or any other company. No assurance can be made to the accuracy or completeness of the information provided herein. Furthermore, no assurance can be made to the accuracy of other sites to which this site links, or of sites that link to this site. No information on this site or obtained from Advocure members should be regarded as medical advice, which should always be sought by consultation with a medical professional.