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New hope for NF1 patients: drug trial targets painful tumors

NCT ID NCT04924608

Disease control Sponsor: AstraZeneca Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the drug selumetinib in adults with neurofibromatosis type 1 (NF1) who have painful, inoperable nerve tumors called plexiform neurofibromas. About 145 participants will receive either selumetinib or a placebo to see if the drug can shrink the tumors and reduce pain. The goal is to control the disease and improve quality of life, not to cure it.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

145 people

The number who actually took part.

Started

Nov 2021

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

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.

Ages

18 years and older

Sex

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN * At least one inoperable target PN measurable by volumetric MRI analysis * Chronic target PN pain score documented for minimum period during screening period * Stable chronic PN pain medication use at enrollment * Adequate organ and marrow function Key Exclusion Criteria: * 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) * 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 * 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 * Ophthalmological findings/conditions including intraocular pressure \> 21 mmHg, RPED/CSR or RVO * Prior exposure to MEK inhibitors

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Conditions

The condition(s) this trial relates to.

neurofibromatosis type 1 plexiform neurofibroma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Neurofibromatosis 1 Plexiform neurofibroma (PN)

Contacts and locations

Locations

  • Research Site

    Gainesville, Florida, 32610, United States

  • Research Site

    Rockville, Maryland, 20852, United States

  • Research Site

    St Louis, Missouri, 63156, United States

  • Research Site

    Commack, New York, 11725, United States

  • Research Site

    Melbourne, 3000, Australia

  • Research Site

    St Leonards, 2065, Australia

  • Research Site

    Porto Alegre, 90035-903, Brazil

  • Research Site

    Ribeirão Preto, 14051-140, Brazil

  • Research Site

    São Paulo, 045202-001, Brazil

  • Research Site

    Toronto, Ontario, M5G 2C4, Canada

  • Research Site

    Montreal, Quebec, H4A 3J1, Canada

  • Research Site

    Beijing, 100070, China

  • Research Site

    Beijing, 100730, China

  • Research Site

    Guangzhou, 510060, China

  • Research Site

    Shenyang, 110001, China

  • Research Site

    Créteil, 94000, France

  • Research Site

    Lyon, 69008, France

  • Research Site

    Toulouse, 31059, France

  • Research Site

    Hamburg, 20246, Germany

  • Research Site

    Tübingen, 72076, Germany

  • Research Site

    Würzburg, 97080, Germany

  • Research Site

    Milan, 20133, Italy

  • Research Site

    Naples, 80131, Italy

  • Research Site

    Roma, 00165, Italy

  • Research Site

    Minatoku, 105-8471, Japan

  • Research Site

    Nagoya, 466-8560, Japan

  • Research Site

    Shinjuku-ku, 160-8582, Japan

  • Research Site

    Bydgoszcz, 85-094, Poland

  • Research Site

    Moscow, 115522, Russia

  • Research Site

    Moscow, 125412, Russia

  • Research Site

    Badalona, 08916, Spain

  • Research Site

    Madrid, 28041, Spain

  • Research Site

    London, SE1 9RT, United Kingdom

  • Research Site

    Manchester, M20 4BX, United Kingdom

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