A Study to Assess the MEK Inhibitor Mirdametinib (PD-0325901) in Patients with Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

Overview

About this study

The purpose of this study is to evaluate PD-0325901 in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive PD-0325901.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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 end of treatment if ≥ 18 years of age. 
  • Participant has adequate organ and bone marrow function.
  • Participant can swallow capsules whole if the capsule dosage form is being utilized. This criterion does not apply if participant is utilizing the dispersible tablet dosage form of study treatment;

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 PD-0325901. 
  • Participant has received NF1 PN-targeted therapy within 45 days. 
  • 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.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Dusica Babovic-Vuksanovic, M.D.

Contact us for the latest status

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Radhika Dhamija, M.B.B.S.

Contact us for the latest status

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

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