Overview | Neurofibromatosis Type 1 | Neurofibromatosis Type 2 | Schwannomatosis | Multi-type Trials | Clinical Trials Consortium

Neurofibromatosis (NF) refers to a group of three genetically distinct disorders that have a spectrum of clinical manifestations. The disorders, NF1, NF2, and Schwannomatosis, can all lead to tumor growth. Other manifestations include learning deficits and cognitive disorders, skin conditions, visual impairments, nervous system disorders and neurological symptoms, vascular disease, musculoskeletal disorders, malignancies, hearing impairments, and chronic pain. Treatments for NF currently include surgery, radiation, and chemotherapy, but several clinical trials are underway to identify more treatment options.

The Congressionally Directed Medical Research Programs’ Neurofibromatosis Research Program (NFRP) is funding several efforts focused on finding better treatments through two main mechanisms, the Clinical Trial Award and the NF Clinical Trials Consortium. A summary of the trials funded to date are found in the tabs on this page.

Neurofibromatosis Type 2 Trials

Updated November 2020

NF160065: Prospective, Randomized, Placebo-Controlled Phase 2 Trial of Aspirin for Vestibular Schwannomas
Summary: This study is a prospective phase 2 trial with the objectives to evaluate whether the administration of aspirin can prevent growth of vestibular schwannomas, as well as determine whether biomarkers from patients’ blood would be able to predict a response to aspirin treatment.
Status: Recruiting
ClinicalTrials.gov Identifier: NCT03079999

NF160012: Open-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults with Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas
Summary: This Phase 2 trial will determine the response rate to crizotinib in children and adult NF2 patients with vestibular schwannomas, and determine safety and tolerability of treatment in these cohorts.  This trial is being performed by the NF Clinical Trials Consortium.
Status: Recruiting
ClinicalTrials.gov Identifier: NCT04283669

NF150056: Phase 2 Study of AZD2014, a Dual mTORC1/mTORC2 Inhibitor, for NF2 Patients with Progressive or Symptomatic Meningiomas
Summary: The goal of this Phase 2 clinical trial is to test the effectiveness of the drug, AZD2014, in treatment of progressive or symptomatic meningiomas. Patient response will be correlated with biomarkers in tumor tissue and blood in an effort to determine markers that will predict efficacy of the drug in future patients.
Status: Active, not recruiting
ClinicalTrials.gov Identifier: NCT02831257

NF120074: Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
Summary: This phase 0, multi-center, proof of concept study assesses the suppression of p-AKT in vestibular schwannoma and meningioma by AR-42, a novel histone deacetylase inhibitor.  The study was amended recently to include patients with cutaneous schwannoma.
Status: Active, not recruiting
ClinicalTrials.gov Identifier: NCT02282917

NF110052: Bevacizumab for NF2-Related Progressive Vestibular Schwannomas
Summary: This study is an open-label, Phase II clinical trial to evaluate bevacizumab in children and young adults with NF2 and progressive vestibular schwannomas that are poor candidates for standard treatment with surgery or radiation. The primary objective of this study is to determine the hearing response rate after beginning treatment with bevacizumab for symptomatic vestibular schwannomas in children and young adults with NF2. This trial was performed by the NF Clinical Trials Consortium.
Status: Active, not recruiting
ClinicalTrials.gov Identifier: NCT01767792
Outcome: High-dose and standard-dose bevacizumab seem to be similarly effective for treatment of patients with NF2 with hearing loss. Unlike adults, pediatric participants did not experience tumor shrinkage. However, adult and pediatric cohorts reported similar improvement in QOL. Treatments incorporating bevacizumab should be considered for children with NF2.
Publications:Plotkin SR, Duda DG, Muzikansky A, Karajannis MA, Allen J, Blakeley J, Rosser T, Campian JL, Clapp DW, Fisher MJ, Tonsgard J, Ullrich NJ, Thomas C, Cutter G, Korf B, Packer R.  Multicenter prospective phase II and biomarker study of high dose bevacizumab as induction therapy in NF 2 patients with progressive vestibular schwannoma: differential response in pediatric versus adult patients. J Clin Oncol, 37(35):3446-3454, 2019.  PMID:31626572

NF110054: RAD001 Phase II Trial for Neurofibromatosis Type 2-Related Vestibular Schwannoma
Summary: This trial aims to determine whether administration of RAD001 will affect vestibular schwannoma growth. In addition, the trial will assess the efficacy and safety of RAD001 in patients, as well as the effect on hearing function in patients with NF2.
Status: Active, not recruiting
ClinicalTrials.gov Identifier: NCT01345136

NF080100: Phase 2, OPEN-Label, Activity, Pharmacodynamic, Pharmacokinetic, and Safety Study of PTC299 in Patients With Neurofibromatosis Type 2
Summary: This phase 2 trial sought to assess the safety, efficacy, and pharmacodynamics activity of PTC299 in patients with neurofibromatosis type 2. The study will assess 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 neurofibromatosis type 2.
Status: Terminated
ClinicalTrials.gov Identifier: NCT00911248