What side effects are associated with this type of intervention?

MEK inhibitors, such as Mirdametinib, are used in the treatment of Neurofibromatosis type 1-associated plexiform neurofibromas. Common side effects of MEK inhibitors include rash, diarrhea, nausea, fatigue, and peripheral edema. More severe adverse events can include serious skin reactions, cardiomyopathy, ocular toxicity, and interstitial lung disease. Patients undergoing treatment with MEK inhibitors should be closely monitored for these potential side effects.

What prior FDA approvals exist?

The FDA has approved selumetinib, a MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas. This approval is significant as it provides a targeted therapy option for patients with NF1, similar to the investigational drug Mirdametinib, which is also a MEK inhibitor being studied for its efficacy in treating NF1-associated plexiform neurofibromas.

What other types of research exist?

Promising alternatives to Mirdametinib for Neurofibromatosis patients include other MEK inhibitors like Selumetinib, which has shown efficacy in clinical trials for NF1-associated plexiform neurofibromas. Additionally, therapies targeting the PI3K/AKT/mTOR pathway, such as Everolimus, may also be considered due to their role in regulating cellular growth and survival.

← Back to Search

MEK Inhibitor

MEK Inhibitor for Neurofibromatosis (ReNeu Trial)

Phase 2

Waitlist Available

Led By Christopher L Moertel, MD

Research Sponsored by SpringWorks Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant has a PN that cannot be completely surgically removed.

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

Must not have

Participant has active bacterial, fungal or viral infection

Participant has breast cancer within 10 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests mirdametinib, a medication taken by mouth, for patients with NF1-related tumors that can't be surgically removed. The drug works by blocking specific proteins to stop or shrink the tumors. Mirdametinib has shown significant and lasting decreases in pain and partial responses in NF1-related plexiform neurofibromas.

Who is the study for?

This trial is for people with a condition called neurofibromatosis type-1 (NF1) and associated inoperable plexiform neurofibromas causing significant problems. Participants must have proper organ function, be willing to undergo tumor biopsies if they're adults, and their tumors should be measurable by MRI. They can't join if they've had certain recent treatments or severe health issues including heart disease, liver disease, infections, some cancers within the last 5 years, or are unable to tolerate MRI.

What is being tested?

The study tests mirdametinib (PD-0325901), given orally as a capsule or tablet, on patients with NF1-associated plexiform neurofibromas that cannot be surgically removed. Everyone in the study will receive this medication and may continue into a long-term follow-up phase to assess ongoing effects.

What are the potential side effects?

While not explicitly listed here, MEK inhibitors like mirdametinib can cause side effects such as skin rash, diarrhea, fatigue and swelling of extremities. There might also be risks related to abnormal blood tests reflecting organ functions which will need monitoring during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My peripheral nerve tumor cannot be fully removed by surgery.

Select...

I am willing to have a tumor biopsy before and after treatment and I am 18 or older.

Select...

My nerve tumor cannot be fully removed by surgery.

Select...

I am willing to have a tumor biopsy before and after treatment and I am 18 or older.

Select...

I have a nerve problem that significantly affects my daily life.

Select...

I have a confirmed diagnosis of neurofibromatosis type 1 (NF1) or a documented NF1 mutation.

Select...

I have a nerve problem that significantly affects my daily life.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I currently have an active infection.

Select...

I was diagnosed with breast cancer within the last 10 years.

Select...

I have been treated with mirdametinib or similar drugs.

Select...

I have had radiation therapy in the last 6 months or to my eye area at any time.

Select...

I cannot have or tolerate an MRI scan.

Select...

I have a condition that affects how my body absorbs medication.

Select...

I have liver disease or abnormal liver function.

Select...

I have a history of eye problems, including retinal issues or glaucoma.

Select...

I need treatment for my optic or low-grade glioma.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Complete or partial response rate at the end of the Treatment Phase compared to baseline. Partial response is defined as a ≥ 20% reduction in target tumor volume.

Secondary study objectives

Change from Baseline in pain as measured by the Numeric Rating Scale-11 (NRS-11).

Change from Baseline in pain as measured by the Pain Interference Index (PII).

Change from Baseline on quality of life (QOL) as measured by the Pediatric Quality of Life Inventory (PedsQL), Acute version.

+2 more

Other study objectives

Acceptability of the dispersible tablet formulation as measured by the Pediatric Oral Medicine Acceptability Questionnaire (P-OMAQ)

Change from Baseline in PN-associated disfigurement using standardized photography, centrally reviewed.

Change from Baseline in endurance.

+7 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Mirdametinib (PD-0325901)Experimental Treatment1 Intervention

Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Neurofibromatosis, such as MEK inhibitors like Mirdametinib, work by targeting specific pathways involved in tumor growth and survival. MEK inhibitors block the MEK enzyme, which is part of the MAPK/ERK pathway, crucial for cell division and growth. Other treatments include bevacizumab, an anti-angiogenic agent that inhibits blood vessel formation in tumors, and everolimus, an mTOR inhibitor that disrupts cellular growth and metabolism pathways. These treatments are significant for Neurofibromatosis patients as they target the underlying biological mechanisms driving tumor growth, potentially reducing tumor size and progression, and improving quality of life.

Network meta-analyses for EGFR mutation-positive non-small-cell lung cancer: systematic review and overview of methods and shortcomings.Pathology and systemic therapy of non-clear cell renal cell carcinoma: an overview.Applied Precision Cancer Medicine in Neuro-Oncology.