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Neurofibromatosis Clinical Trials

Researchers at Johns Hopkins and around the world are investigating ways to slow the growth of NF-related tumors and prevent them from forming altogether.

Although there is not yet a cure for either neurofibromatosis or schwannomatosis, some patients may benefit from clinical trials. Follow the links below to learn more about the various studies available.

Featured Clinical Trials

Neoadjuvant Nivolumab Plus Ipilimumab for Newly Diagnosed Malignant and Pre-malignant Peripheral Nerve Sheath Tumors (MPNST)
Contact: Stella Krawiec (410-502-1962)
Principle Investigator: Jaishri Blakeley, M.D.
Objective: The primary purpose is to explore whether two drugs (nivolumab and ipilimumab) that are approved by the FDA to treat other cancers are safe and feasible to give prior to standard therapy (surgery, chemotherapy or radiation therapy) in patients with Neurofibromatosis Type 1 (NF1) and newly diagnosed pre-malignant and malignant peripheral nerve sheath tumors (MPNST).
Eligibility Criteria: Eligible patients must have histologically confirmed diagnosis of ANNUBP (atypical neurofibromatous neoplasms of uncertain biologic potential), low grade MPNST or high grade MPNST. Patients may have plexiform neurofibroma or other tumors such as optic pathway glioma, other low-grade glioma or other neoplasm in addition to the ANNUBP, low grade MPNST or high grade MPNST that is stable (has not required treatment in the last 12 months and is not anticipated to need treatment in the next 12 months).

Browse Other Trials on

Find additional clinical trials conducted at Johns Hopkins for patients with neurofibromatosis and related conditions.


Patient Registries

Patient registries help NF researchers better understand the disease. Volunteers opt-in to share information with experts across a secure database. Researchers use that data to study disease trends and treatment effectiveness. 

Schwannomatosis Registry
(for patients with schwannomatosis)

This database is maintained with the vision of bringing together patients with schwannomatosis who want to help researchers who are sponsoring trials. By connecting patients and researchers more effectively, the registry seeks to better understand schwannomatosis and uncover helpful treatment options.

NF Registry
(for patients with NF1, NF2 or schwannomatosis)

If you are an adult with NF or the parent or guardian of a child with NF, joining the NF Registry is an easy way to get involved and contribute to more effective treatments. The NF Registry, created by the Children's Tumor Foundation, is a listing of volunteers for participation in clinical trials, and those willing to share information. Researchers use secure methods to identify trends and characteristics of NF incidence and clinical course.

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