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Last-Resort drug access opens for rare tumor patients

NCT ID NCT05028166

Disease control Sponsor: SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany 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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 program provides mirdametinib to individual patients with serious, life-threatening conditions like neurofibromatosis type 1 tumors or other MAP-K pathway diseases who have no other treatment options and cannot join a clinical trial. Each case requires approval from the company, ethics board, and health authorities. The goal is to control the disease when standard treatments have failed.

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.

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

2 years and older

Sex

Anyone

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: * Patient has a serious, debilitating or life-threatening medical condition that cannot be treated satisfactorily with an authorized medicinal product or other available standard treatment options, or all other treatment options have been exhausted. * Patient does not qualify to participate in an ongoing clinical trial.

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Conditions

The condition(s) this trial relates to.

histiocytic and dendritic cell neoplasm histiocytic sarcoma neurofibromatosis plexiform neurofibroma

As listed by the trial registrant

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

Histiocytic neoplasm Neurofibromatosis type 1-associated plexiform neurofibromas Other map-K pathway driven diseases

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  2. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

More trials for these conditions

Other studies related to the condition(s) this trial covers.