Neurological

Neurofibromatosis Type 2

Also known as NF2, bilateral acoustic neurofibromatosis, MISME syndrome, NF2 merlin deficiency

Neurofibromatosis type 2 (NF2) is caused by mutations in the NF2 tumor suppressor gene encoding merlin (schwannomin), a cytoskeletal protein. The hallmark is bilateral vestibular schwannomas causing progressive hearing loss, tinnitus, and b

ORPHA:637 ↗Gene NF2Prevalence 1-9 per 100,000 (Orphanet)Onset Adolescent, AdultAutosomal dominant genetic

16

studies recruiting now

as of 7 Sept 2026

94

studies registered in total

as of 7 Sept 2026

3

countries with a recruiting site

as of 7 Sept 2026

23 Feb 2022

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 16 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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About Neurofibromatosis Type 2

Neurofibromatosis type 2 (NF2) is caused by mutations in the NF2 tumor suppressor gene encoding merlin (schwannomin), a cytoskeletal protein. The hallmark is bilateral vestibular schwannomas causing progressive hearing loss, tinnitus, and balance problems. Patients also develop meningiomas, ependymomas, and other schwannomas throughout the nervous system. Bevacizumab has shown benefit for vestibular schwannoma growth; FAK inhibitors and MEK inhibitors are in active trials.

Common clinical features

Bilateral vestibular schwannomasHearing loss and tinnitusBalance problemsMeningiomasSpinal ependymomasFacial nerve palsyCataracts

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

1 approved treatment and 1 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Selumetinib Sulfate (Koselugo)
Phase 3Sirolimus (Fyarro)

Before you apply

Things trial teams commonly ask about for Neurofibromatosis Type 2. Not eligibility rules; those are set by each study.

  • Tumor growth rate on serial MRI (typically requiring 2 scans 6 months apart) is a standard eligibility criterion
  • Hearing assessment (audiogram and word recognition scores) at baseline is required — hearing preservation is a key trial endpoint
  • Bevacizumab treatment history and response must be documented for anti-angiogenic and alternative therapy trials
  • NF2 severity scale (Manchester) and Karnofsky performance status are used for trial stratification

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).