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Open-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults with Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas


To estimate the best objective volumetric response rates to crizotinib in pediatric and adult NF2 patients with VS.


Crizotinib (Xalkori®), a small molecule kinase inhibitor, reduces NF2 schwannoma cell growth and tumor formation in preclinical schwannoma models via inhibition of FAK1. It is an oral drug approved by the United States Food and Drug Administration (FDA) for the treatment of other types of tumors.


  • NF2 patients ≥6 years of age on day 1 of treatment
  • Subjects must have a target VS with the following qualities:
    • volumetrically measurable and ≥0.75 ml in size
    • volumetric evidence of progression over the past 18 months (defined as ≥20% annualized increase in volume)


Subjects with Neurofibromatosis Type 2 (NF2) and progressive vestibular schwannoma (VS) will be treated with crizotinib administered orally. Crizotinib will be taken continuously until disease progression or unacceptable toxicity, in continuous treatment cycles of 28 days each, for a maximum of 12 cycles. Clinical response will be assessed by MRI (volumetrics, primary objective) and audiology at the end of every 3rd cycle. Subjects with volumetric tumor progression will be taken off protocol. Patients who complete 12 cycles of treatment without disease progression, but within the following 24 weeks show subsequent disease progression (defined as >20% increase in target tumor volume compared to off-treatment volume), will be eligible for re-treatment on study for up to 48 additional weeks, provided they still meet study eligibility criteria.


  • Matthias A. Karajannis, MD, MS (Chair)
  • Phioanh [Leia] Nghiemphu, MD (Co-Chair)
  • Scott R. Plotkin, MD, PhD (Co-Chair)


Enrollment is on hold.


None at this time.