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Evaluating the effects of selumetinib in children with neurofibromatosis type 1

Observational Study of the Effectiveness and Safety of the MEK 1/2 Inhibitor (MEKi) Koselugo (Selumetinib) in Pediatric Patients With Symptomatic, Inoperable, Neurofibromatosis Type 1 (NF1)-Associated Plexiform Neurofibromas (PN)

Observational AstraZeneca · NCT06175637

This study is testing if a new drug called selumetinib can help children with neurofibromatosis type 1 and inoperable tumors feel better and stay safe over two years.

Quick facts

Study typeObservational
Enrollment400 (estimated)
Ages3 Years to 16 Years
SexAll
SponsorAstraZeneca Industry-sponsored
Drugs / interventionsselumetinib, chemotherapy, radiation
Locations34 sites (Beijing, Beijing and 33 other locations)
Trial IDNCT06175637 on ClinicalTrials.gov

What this trial studies

This observational study focuses on pediatric patients aged 3 to 16 years diagnosed with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). Conducted across approximately 12 centers in China, the study aims to enroll 80-100 participants who will begin treatment with selumetinib after consent. Patients will be monitored for 24 months following their first dose to assess the drug's effects and safety. The study seeks to enhance understanding of NF1-PN characteristics and treatment patterns in this population.

Who should consider this trial

Good fit: Ideal candidates are children aged 3 to 16 years with a confirmed diagnosis of NF1 and symptomatic, inoperable plexiform neurofibromas.

Not a fit: Patients with evidence of malignant peripheral nerve sheath tumors or those who have previously received treatment with specific inhibitors may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide insights into effective treatment options for children suffering from NF1-related plexiform neurofibromas.

How similar studies have performed: While this approach is novel in the context of pediatric NF1-PN, similar studies have shown promise in evaluating treatments for other conditions.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* (1) 3≤ age ≤16 years old at the time of study enrollment; (2) Diagnosed with NF1 as per the revised 2021 Guidelines; (3) Have symptomatic, inoperable PN: (4) Intend to use selumetinib after enrollment; (5) Able to give written informed consent. Data collection must only be done after obtaining written informed consent. Patient or patient's parent/legal guardian must be willing and able to give written informed consent. Parent or legal guardian consent is required in the assent process with appropriate documentation. Mandatory provision of signed and dated parent/legal guardian consent for the study along with the pediatric assent form, when applicable.

Exclusion Criteria:

* (1) Evidence of MPNST, prior malignancy or other cancer requiring treatment with chemotherapy or radiation therapy; (2) A life-threatening illness, medical condition, or organ system dysfunction; (3) Have had prior treatment with a MEKi, Ras or Raf inhibitor; (4) Patients currently participating in any clinical trials at the time of enrollment or initiation of selumetinib.

Where this trial is running

Beijing, Beijing and 33 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Neurofibroma, Plexiform
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.