Neurofibromatosis Type 1-Associated Plexiform NeurofibromasNCT05028166
Individual Patient Compassionate Use of Mirdametinib
A study testing Mirdametinib (MEK Inhibitor) in people with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas and Histiocytic Neoplasm, run by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany at 2 sites in 2 countries.
Trial summary
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
- What is being tested?
- Mirdametinib (MEK Inhibitor)
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 2 sitesin 2 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
2 sites in 2 countries.
Contact Early Access contact
2 sites in 2 countries
United States1 site
- Available
Please Contact U.S. Medical Information
Billerica, Massachusetts, 01821
US Medical Information, 888-275-7376, eMediUSA@emdserono.com
Germany1 site
- Available
Please Contact the Communication Center
Darmstadt, 64293
Communication Center, +49 6151 72 5200, service@emdgroup.com
About this study
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
- Conditions
- Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
- Being tested
- Mirdametinib (MEK Inhibitor)
- Keywords
- RASopathies, NF1, Neurofibromatosis, Plexiform Neurofibromas, Langerhans
- Medical subject headings
- Neurofibromatoses, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mirdametinib (MEK Inhibitor)Drug
Patients will receive oral mirdametinib.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1-Associated Plexiform NeurofibromasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05028166
- Sponsor's protocol ID
- MEK-NF1-701
- Status last verified
- Sep 2026
- First submitted
- 24 Aug 2021
- Last updated
- 5 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individual Patient Compassionate Use of Mirdametinib
- ClinicalTrials.gov ID
- NCT05028166
- Acronym / sponsor ID
- MEK-NF1-701
- Conditions
- Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
- Keywords
- RASopathies, NF1, Neurofibromatosis, Plexiform Neurofibromas, Langerhans
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- First posted
- 31 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mirdametinib (MEK Inhibitor)Patients will receive oral mirdametinib. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- Trial Awareness and Transparency websiteLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Contact Early Access contact
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mirdametinib (MEK Inhibitor) in people with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas and Histiocytic Neoplasm, run by SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact US Medical Information (888-275-7376, eMediUSA@emdserono.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2026.