Learn more about neurofibromatosis (NF) syndromes and our research to help us find better treatments.
Neurofibromatosis Clinical Team
Our multidisciplinary team develops and conducts clinical trials to find better treatments for children and adolescents with neurofibromatosis.
Neurofibromatosis Clinical Team
The POB NF team has been studying NF and NF-related tumors for over 25 years. In 2020, years of research culminated in the FDA approval of selumetinib, the first FDA approved drug for children 2 years and older with NF1 and NF1-related inoperable nerve tumors. Today, their research efforts continue to expand to improve the lives of children and adults with NF.
Patients & Providers
Learn more about how to participate in our clinical trials and find resources for NF management and care.
Contact our team at 301-594-9976 or baldwinam@mail.nih.gov if you have questions or need help finding a clinical trial.
Clinical Trials
- Phase II Trial of the MEK inhibitor Selumetinib in Adults with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
- Phase II Trial of the MEK Inhibitor Selumetinib in Combination with the mTOR Inhibitor Sirolimus for Patients with Unresectable or Metastatic Malignant Peripheral Nerve Sheath Tumors
- Phase I/II Study of the Cyclin Dependent Kinase (CDK) 4/6 Inhibitor Abemaciclib for Neurofibromatosis Type 1 (NF1) Related Atypical Neurofibromas
- Pilot Study of the MEK 1/2 Inhibitor for Adults with Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (cNF)
- Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement from Photographs of Subjects with Neurofibromatosis Type 1 on a Phase II Clinical Trial
- RASopathies Study: This study seeks to learn more about the development of tumors in individuals with a RASopathy (excluding NF1).