MEK Inhibitor for Neurofibromatosis
(ReNeu Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called mirdametinib (a MEK inhibitor) for individuals with neurofibromatosis type 1 (NF1), a genetic disorder that can cause tumors known as plexiform neurofibromas (PNs). The goal is to determine if this medication can reduce symptoms in those with tumors that cannot be surgically removed. Participants will take mirdametinib and may continue with long-term follow-up. This trial may suit individuals with NF1 who have troublesome tumors that cannot be fully removed by surgery and are willing to undergo MRIs and biopsies for study purposes. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you have received NF1 PN-targeted therapy within 45 days, you may not be eligible to participate.
Is there any evidence suggesting that mirdametinib (PD-0325901) is likely to be safe for humans?
Research has shown that mirdametinib, a treatment for neurofibromatosis type 1 (NF1) with plexiform neurofibromas, has been studied for safety. In a previous study, adults taking mirdametinib experienced some side effects, but these were usually manageable. Common side effects included fatigue, nausea, and diarrhea, while serious side effects were less common.
Importantly, the FDA has already approved mirdametinib for treating both adults and children with NF1, indicating it has passed several safety checks for this condition. This approval suggests the treatment is considered safe for these groups. Prospective trial participants might find this existing information reassuring.12345Why do researchers think this study treatment might be promising?
Mirdametinib is unique because it targets a specific pathway called the MEK/ERK pathway, which is often overactive in neurofibromatosis. Unlike standard treatments that might focus on managing symptoms or surgical options, Mirdametinib offers a targeted approach by directly inhibiting this pathway, potentially slowing or stopping the growth of tumors associated with the condition. Researchers are excited about this treatment because it could provide a more effective and less invasive option for patients, improving their quality of life by addressing the root cause of tumor growth.
What evidence suggests that mirdametinib might be an effective treatment for neurofibromatosis?
Research has shown that mirdametinib, the treatment under study in this trial, may help treat plexiform neurofibromas (PNs) associated with neurofibromatosis type-1 (NF1). In one study with 19 patients, about 42% experienced tumor shrinkage. Another study with 50 children found that 66% responded positively to the treatment. On average, it took about 5.4 months for half of the participants to notice these changes. Mirdametinib has been well-tolerated, with most people not experiencing serious side effects. These findings suggest that mirdametinib could effectively manage NF1-PNs.24567
Who Is on the Research Team?
Christopher Moertel
Principal Investigator
University of Minnesota
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive mirdametinib (PD-0325901) for the treatment of symptomatic inoperable neurofibromatosis type-1 associated plexiform neurofibromas
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up (optional)
Eligible participants may continue in a long-term follow-up phase
What Are the Treatments Tested in This Trial?
Interventions
- Mirdametinib (PD-0325901)
Find a Clinic Near You
Who Is Running the Clinical Trial?
SpringWorks Therapeutics, Inc.
Lead Sponsor