Real-World study tracks Selumetinib's impact on NF1 tumors in children
NCT ID NCT05891847
First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time
Summary
This study observes children aged 3 to 18 with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas and are starting treatment with selumetinib. Researchers will track how well the drug shrinks or stabilizes tumors, how long patients stay on treatment, and any side effects. The goal is to see how selumetinib works in everyday medical practice in Russia.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- selumetinib
- What this could lead to
- If successful, this study could confirm that selumetinib effectively shrinks or stabilizes these tumors in children with NF1 outside of controlled trials.
- What could go wrong
- This is an observational study, not a controlled experiment, so results may be less reliable. Selumetinib can cause side effects like nausea, diarrhea, and skin issues.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
150 people
The number who actually took part.
- Started
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Mar 2023
- Finished
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Jul 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
- Ages
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3 to 18 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * NF1 diagnosed (according to the criteria for evaluating NF1), * Symptomatic inoperable PN, * Patients receiving therapy with selumetinib. For newly treated patients - registration on the first day of starting treatment with selumetinib, * Age at starting of selumetinib treatment ≥ 3 to ≤ 18 years old (included in the study or started the therapy at the age of 18 years old), * Patients whose parents or guardians have signed a consent form. Exclusion Criteria: * Patients currently participating in an interventional study/clinical trial, * Patients are excluded if there is evidence of an optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy, * Patients who receive other antitumor treatment options on optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer. If the patient on selumetinib treatment develop optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer, he can continue selumetinib therapy +\\- other treatment options on physician decision, * Female patients above 12 age who are pregnant /plan to become pregnant or at breast feeding.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Research Site
Moscow, Moscow, 123112, Russia
-
Research Site
Moscow, Russia
-
Research Site
Novosibirsk, Russia
-
Research Site
Saint Petersburg, Russia
-
Research Site
Yekaterinburg, Russia
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