Individual Patient Compassionate Use of Mirdametinib

NCT05028166 | Unknown

• 1 other identifier: MEK-NF1-701
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Neurofibromatosis Type 1-Associated Plexiform Neurofibromas; Histiocytic Neoplasm; Other MAP-K Pathway Driven Diseases

Keywords

RASopathies; NF1; Neurofibromatosis; Plexiform Neurofibromas; Langerhans

Interventions

Mirdametinib (MEK Inhibitor) DRUG

Patients will receive oral mirdametinib.

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• SpringWorks Therapeutics, Inc.: lead

MeSH Terms

Conditions

Neurofibromatoses; Neurofibroma, Plexiform

Interventions

mirdametinib

Condition Hierarchy (Ancestors)

Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Peripheral Nervous System Diseases; Neuromuscular Diseases

Central Study Contacts

US Medical Information

CONTACT

888-275-7376; eMediUSA@emdserono.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2021
• First Posted: August 31, 2021
• Last Updated: March 27, 2026

Record last verified: 2026-03