For Healthcare Professionals

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas


About the study

A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


Key Inclusion Criteria:

  1. Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN
  2. At least one inoperable target PN measurable by volumetric MRI analysis
  3. Chronic target PN pain score documented for minimum period during screening period
  4. Stable chronic PN pain medication use at enrollment
  5. Adequate organ and marrow function


Key Exclusion Criteria:

  1. Confirmed or suspected malignant glioma or MPNST (low grade glioma, including optic glioma not requiring systemic therapy or radiation therapy are exempt from this exclusion)
  2. History of malignancy except for malignancy treated with curative intent with no known active disease ≥ 5 years before the first dose of study intervention and of low potential risk for recurrence
  3. Clinically significant cardiovascular disease, including inherited coronary disease, acute coronary syndrome within 6 months prior to enrollment, uncontrolled angina, symptomatic heart failure, cardiomyopathy, severe valvular heart disease, abnormal LVEF and uncontrolled hypertension
  4. Ophthalmological findings/conditions including intraocular pressure > 21 mmHg, RPED/CSR or RVO
  5. Prior exposure to MEK inhibitors

pin location

Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Neurofibromatosis 1,Plexiform Neurofibroma (PN)

Age (in years)



Phase 3

Participants needed


Est. Completion Date

May 22, 2025

Treatment type



AstraZeneca identifier


Study number


Understanding Clinical Trials

Get answers to your questions about clinical trials.What is a clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?


Sign up to save your favorites, 
receive newsletters, resources, and alerts 
about clinical trials related to your conditions of interest.