Trial Search Results

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

SpringWorks Therapeutics, Inc.

Stanford Investigator(s):

Intervention(s):

  • Drug: Mirdametinib (PD-0325901) oral capsule or dispersible tablet

Phase:

Phase 2

Eligibility


Key Inclusion Criteria:

   - Participant has documented NF1 mutation or a diagnosis of neurofibromatosis type 1
   (NF1) using National Institute of Health (NIH) Consensus Conference criteria inclusive
   of the presence of a plexiform neurofibroma (PN).

   - Participant has a PN that is causing significant morbidity.

   - Participant has a PN that cannot be completely surgically removed.

   - Participant has a target tumor that is amenable to volumetric MRI analysis.

   - Participant is willing to undergo a tumor biopsy pre and post treatment if ≥ 18 years
   of age.

   - Participant has adequate organ and bone marrow function.

Key Exclusion Criteria:

   - Participant has abnormal liver function or history of liver disease.

   - Participant has lymphoma, leukemia or any malignancy within the past 5 years (except
   for resected basal/squamous skin carcinomas without metastases within 3 years).

   - Participant has breast cancer within 10 years.

   - Participant has active optic glioma or other low-grade glioma requiring treatment.

   - Participant has abnormal QT interval corrected or other heart disease within 6 months.

   - Participant has a history of retinal pathology, risk factors for retinal vein
   occlusion or has a history of glaucoma.

   - Participant has known malabsorption syndrome or gastrointestinal conditions that would
   impair absorption of mirdametinib (PD-0325901).

   - Participant has received NF1 PN-targeted therapy within 45 days.

   - Participant previously received or is currently receiving therapy with mirdametinib
   (PD-0325901) or any other MEK1/2 inhibitor.

   - Participant has received radiation therapy within 6 months or has received radiation
   to the orbit at any time.

   - Participant is unable to undergo or tolerate MRI.

   - Participant has active bacterial, fungal or viral infection.

   - Participant has experienced other severe acute or chronic medical or psychiatric
   conditions within 1 year.

Ages Eligible for Study

2 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Erica Velasco, SC
Not Recruiting