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New study tracks Long-Term safety of NF1 drug in kids

NCT ID NCT05388370

Knowledge-focused 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 25, 2026 · Updated 2 times

Summary

This study monitors the safety of selumetinib in children aged 3 to 18 with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Researchers will track potential side effects on the heart, bones, muscles, liver, eyes, and puberty. The goal is to better understand the drug's risks in real-world use across Europe and Israel.

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

124 people

The number who actually took part.

Started

May 2022

Expected to finish

May 2028

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.

Who is studied

The target population for this study are patients with NF1 with symptomatic, inoperable PN who have been prescribed at least 1 dose of selumetinib and who are aged 3 to \< 18 years at the start of selumetinib treatment, except for those patients receiving treatment with a mitogen-activated protein kinase inhibitor before the index date.

Ages

3 to 17 years

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.

Inclusion Criteria: * Have been diagnosed with NF1 with symptomatic, inoperable PN * Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to enrolment into the study (i.e. signature of the ICF) * Are aged 3 years and above, and are \< 18 years of age on the index date * Parent or legal guardian, as required by country-specific regulation, have provided informed consent (unless a country-specific waiver is obtained) Additional Criteria for Nested Prospective Cohort * Are at least 8 years old and * Are prior to attainment of Tanner Stage V on the index date Exclusion Criteria: * Have received treatment with a mitogen-activated protein kinase inhibitor before the index date * Are participating in an interventional study at index date

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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 type 1

Contacts and locations

Locations

  • Research Site

    Vienna, Austria

  • Research Site

    Amiens, France

  • Research Site

    Angers, France

  • Research Site

    Bordeaux, France

  • Research Site

    Lille, France

  • Research Site

    Lyon, France

  • Research Site

    Marseille, France

  • Research Site

    Paris, France

  • Research Site

    Rennes, France

  • Research Site

    Strasbourg, France

  • Research Site

    Toulouse, France

  • Research Site

    Tours, France

  • Research Site

    Vandœuvre-lès-Nancy, France

  • Research Site

    Villejuif, France

  • Research Site

    Dresden, Germany

  • Research Site

    Duisburg, Germany

  • Research Site

    Hamburg, Germany

  • Research Site

    Hanover, Germany

  • Research Site

    München, Germany

  • Research Site

    Tübingen, Germany

  • Research Site

    Petah Tikva, Israel

  • Research Site

    Ramat Gan, Israel

  • Research Site

    Tel Aviv, Israel

  • Research Site

    Florence, Italy

  • Research Site

    Genova, Italy

  • Research Site

    Milan, Italy

  • Research Site

    Padova, Italy

  • Research Site

    Pavia, Italy

  • Research Site

    Roma, Italy

  • Research Site

    Torino, Italy

  • Research Site

    Trieste, Italy

  • Research Site

    Rotterdam, Netherlands

  • Research Site

    Lisbon, Portugal

  • Research Site

    Porto, Portugal

  • Research Site

    Barcelona, Spain

  • Research Site

    Madrid, Spain

  • Research Site

    Málaga, Spain

  • Research Site

    Santiago de Compostela, Spain

  • Research Site

    Seville, Spain

  • Research Site

    Basel, Switzerland

  • Research Site

    Bern, Switzerland

  • Research Site

    Lausanne, Switzerland

  • Research Site

    Sankt Gallen, Switzerland

  • Research Site

    London, United Kingdom

  • Research Site

    Manchester, United Kingdom

  • Research Site

    Newcastle upon Tyne, United Kingdom

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