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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 24 months
Awards & highlights

ReNeu Trial Summary

This trial is testing a new drug, mirdametinib, to see if it can help improve the symptoms of neurofibromatosis type-1 (NF1) - associated plexiform neurofibromas (PNs).

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.Check my eligibility
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.See study design
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.

ReNeu Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My peripheral nerve tumor cannot be fully removed by surgery.
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I am willing to have a tumor biopsy before and after treatment and I am 18 or older.
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My nerve tumor cannot be fully removed by surgery.
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I am willing to have a tumor biopsy before and after treatment and I am 18 or older.
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I have a nerve problem that significantly affects my daily life.
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I have a confirmed diagnosis of neurofibromatosis type 1 (NF1) or a documented NF1 mutation.
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I have a nerve problem that significantly affects my daily life.

ReNeu Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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 outcome measures
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

ReNeu 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

Find a Location

Who is running the clinical trial?

SpringWorks Therapeutics, Inc.Lead Sponsor
12 Previous Clinical Trials
852 Total Patients Enrolled
Christopher L Moertel, MDPrincipal InvestigatorUniversity of Minnesota

Media Library

Mirdametinib (PD-0325901) (MEK Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03962543 — Phase 2
Neurofibromatosis Research Study Groups: Mirdametinib (PD-0325901)
Neurofibromatosis Clinical Trial 2023: Mirdametinib (PD-0325901) Highlights & Side Effects. Trial Name: NCT03962543 — Phase 2
Mirdametinib (PD-0325901) (MEK Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03962543 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants are participating in this research endeavor?

"This research project is no longer taking on volunteers. The trial was initially posted in September 2019 and last updated November 2022, but if you are searching for other trials, 56 studies currently offer enrolment to those with plexiform neurofibroma and 4 studies provide the opportunity to take part in Mirdametinib (PD-0325901) oral capsule or dispersible tablet experiments."

Answered by AI

How many locations are actively conducting this experimentation?

"Mayo Clinic in Rochester, University of Chicago Medical Centers in Chicago and Henry-Joyce Cancer Clinic in Nashville are some of the 54 hospitals that are participating in this medical trial."

Answered by AI

Are there any open slots available for this trial at the present time?

"The particulars of this particular research project, which first appeared on the clinicaltrials.gov website on September 6th 2019 and was most recently updated November 17th 2022, suggest that patient recruitment is not taking place at present; however, there are presently 60 other trials actively seeking participants."

Answered by AI

Are there other clinical investigations which have tested the efficacy of Mirdametinib (PD-0325901) in either a capsule or tablet form?

"Presently, 4 clinical trials are actively researching Mirdametinib (PD-0325901) oral capsule or dispersible tablet; none of these studies have progressed to Phase 3. The majority of the research is taking place in Melbourne, Victoria with 63 different locations participating in testing this medication."

Answered by AI

What risks does Mirdametinib (PD-0325901) oral capsule or dispersible tablet pose to individuals?

"Taking into account the fact that this is a stage 2 trial with safety data but no evidence of efficacy, our experts at Power assigned Mirdametinib (PD-0325901) oral capsule or dispersible tablet a score of 2."

Answered by AI

What is the desired result of this medical trial?

"The principal aim of this 24-month long study is to evaluate the complete or partial response rate at the end of the treatment phase compared against baseline. Partial response is defined as a ≥ 20% reduction in target tumor volume. Secondarily, researchers will measure change from baseline pain interference with activities via Pain Interference Index (PII), duration of response for those that meet criteria for objective responses by means of volumetric MRI review and change from beginning quality of life using Pediatric Quality of Life Inventory (PedsQL) Acute version."

Answered by AI

Who else is applying?

What site did they apply to?
UCLA Oncology Center
What portion of applicants met pre-screening criteria?
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
2019. "MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03962543.
All > Neurofibromatosis Type 1 > Neurofibromatosis
~21 spots leftby Apr 2025
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