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Clinical Trials Pipeline

Discovery and development of NF drug candidates.

Clinical Trials Progress

There are now 67 active clinical trials for NF – three times the number of clinical trials than there were just 10 years ago. The FDA approval of selumetinib (Koselugo) proved to be a beacon for NF researchers, pharma, and biotech companies, who have a renewed interest in NF.


FDA-approved Drug for NF, and more on the horizon

CTF-funded research paved the way to the first FDA-approved drug for NF, Koselugo (selumetinib).


the number of NF Clinical Trials in the past 10 years

There are currently 67 active clinical trials for NF – three times the number of trials than just ten years ago!


years of funding NF research

CTF was founded 45 years ago as the first grassroots organization to support patients with all types of NF.


have joined the NF Registry

The NF Registry is a secure website where people with NF can take an active role in the search for better treatments.


patients each year visit the NF Clinic Network

The NF Clinic Network (NFCN) is dedicated to improving NF clinical care and establishing best practices.

NF Registry

The NF Registry represents the collaborative efforts of patients from around the world diagnosed with NF, including neurofibromatosis type 1 (NF1) and all forms of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN), formerly called neurofibromatosis type 2.

The NF Registry was created in order to accelerate research and therapy development for patients. The database holds the information necessary to determine the prevalence and natural history of the disorders and to assist in identifying candidate patients for a particular study.

Colorful Logo that reads NF Registry

About the NF Registry

The database houses the information necessary to determine the prevalence and natural history of the disorders as well as to assist in the identification of appropriate candidate patients for a particular study, all of whom have agreed to be contacted by CTF about potential beneficial research. Identifiable information for registry participants will never be released by CTF. For centers conducting clinical trials, participants who may be candidates for studies will be contacted by CTF and provided with contact information; if the patient is interested in potential participation in a study, he or she will contact the study center.

Visit NF Registry

Available Projects for Researchers

There are two main ways researchers use NF Registry data:

  1. Recruit patients for an IRB-approved clinical trial or research study.
  2. Request de-identified data maintained in the NF registry for data analysis.

All requests are reviewed by the Data Access Committee, consisting of NF clinicians, patients, and care advocates.

For all requests, please contact Kate Kelts at

Learn More

NF Clinical Trials Consortium

The Neurofibromatosis Clinical Trials Consortium (NFCTC) was established by the Department of Defense Neurofibromatosis Research Program (NFRP) to develop and perform clinical trials for the treatment of NF complications in children and adults. The Consortium is composed of fifteen clinical sites, ten collaborating affiliate sites, and an Operations Center at the University of Alabama at Birmingham under the direction of Dr. Bruce Korf.

This presentation about the NF Clinical Trials Consortium was presented live on June 23, 2023 at the Children’s Tumor Foundation NF Summit in Scottsdale, Arizona.

Platform Trials

CTF is a funding partner with EU-PEARL (EU Patient-cEntric clinicAl tRial pLatforms), a joint project under a European Innovative Medicine Initiative (IMI). This pursuit is building platform trials in which multiple drugs are tested in parallel under the same clinical protocol. This approach allows more efficient identification of potential treatments.

Through this initiative, an NF1 platform trial protocol is being defined and finalized and will be operationalized through CTF and the Global Coalition for Adaptive Research. It will initially focus on treatments for plexiform neurofibromas, cutaneous neurofibromas, optic pathway gliomas (OPG), and non-OPG low-grade gliomas.

Separately, CTF is funding the INTUITT-NF2-SWN trial, which is currently in progress and testing brigatinib and neratinib in patients with NF2-SWN. This initiative is a result of the landmark work of CTF’s visionary Synodos for NF2 research collaboration. Read more on our Progress in NF2-SWN page.

Read More about Platform Trials

Thanks to CTF’s advocacy efforts in Europe, NF was chosen as a prototype for rare diseases by the IMI. The NF component of EU-PEARL is co-lead by CTF and the Erasmus Medical Center in Rotterdam, Netherlands. While a network of European clinics will be the first of many to execute these trials, the established protocols will enable trials such as this to run globally.

In addition to the NF1 protocol, a schwannomatosis platform trial protocol is being developed that will enroll schwannomatosis patients, including NF2-related schwannomatosis patients, and will test the ability of drugs to shrink tumors.

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