Mirdametinib for Melanoma

Overseen By Andrea Davila

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Kevin Kim, MD

Must not be taking: MEK inhibitors

Disqualifiers: Symptomatic brain metastasis, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called mirdametinib, a type of targeted therapy, for individuals with advanced melanoma that has an NF1 mutation. The trial evaluates mirdametinib's effectiveness for patients whose cancer has progressed despite previous treatments, including certain immunotherapies. It also assesses the safety of mirdametinib for these patients. The trial is best suited for individuals whose melanoma continues to grow despite prior immunotherapy and who have an NF1 mutation confirmed by specific genetic testing. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that you should not have had systemic cancer therapy within 28 days before starting the study drug. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that mirdametinib is likely to be safe for humans?

Previous studies have examined the safety of mirdametinib for various conditions. The research shows that most adults taking mirdametinib felt better by the 13th treatment cycle. However, some patients experienced side effects. Nearly all adult patients reported side effects from the treatment, occasionally leading to dosage adjustments or discontinuation. Despite these challenges, the FDA has approved mirdametinib for other conditions, indicating a certain level of safety. Overall, while mirdametinib seems promising, potential participants should consider the possibility of side effects when deciding to join a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Mirdametinib is unique because it targets a specific pathway in melanoma cells known as the MEK pathway, which is crucial for cancer cell growth and survival. Unlike traditional treatments like chemotherapy or immunotherapy, which can have broad and intense effects on the body, Mirdametinib specifically inhibits MEK, potentially leading to fewer side effects and more targeted action against cancer cells. Researchers are excited about Mirdametinib because it offers a new mechanism of action that could be more effective for patients with melanoma that doesn't respond well to current treatments.

What evidence suggests that mirdametinib might be an effective treatment for melanoma?

Studies have shown that mirdametinib can help treat NF1-mutant melanoma, a type of skin cancer. Patients with similar conditions have experienced health improvements with this treatment. Specifically, mirdametinib has reduced the severity of tumor-related symptoms. Research also suggests that mirdametinib can slow disease progression. These findings indicate that mirdametinib may be a promising option for those with advanced NF1-mutant melanoma, especially when other treatments are no longer effective. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for patients with advanced melanoma that has a specific mutation called NF1. Participants must have experienced disease progression while on or after undergoing immunotherapy treatments.

Inclusion Criteria

My melanoma cannot be surgically removed and has an NF1 mutation confirmed by a certified lab.

I've been treated with specific immune therapies unless they were not suitable for me.

Patients must have measurable metastatic disease according to RECIST 1.1

Exclusion Criteria

Patients must not be simultaneously enrolled in any therapeutic clinical trial

Patient who is pregnant or breastfeeding

I do not have an active infection.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive mirdametinib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Safety Monitoring

Descriptive statistics for safety will be presented using NCI CTCAE version 5.0. All on-study AEs and other safety measures will be tabulated.

2 years

What Are the Treatments Tested in This Trial?

Interventions

• Mirdametinib

Trial Overview The study is testing the drug mirdametinib to see if it's effective and safe in treating advanced NF1-mutant melanoma, specifically in cases where the cancer continued to grow despite previous immunotherapy.

How Is the Trial Designed?

Group I: Mirdametinib Experimental Treatment 1 Intervention

Patients receive mirdametinib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Who Is Running the Clinical Trial?

Kevin Kim, MD

Lead Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07237100

Mirdametinib in Patients With Advanced NF1-mutant ... The purpose of this study is to test if mirdametinib is safe and effective in improving disease status and/or delaying progression of disease.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12592225/

Combination of the Novel RAF Dimer Inhibitor Brimarafenib ... While targeted kinase inhibitors have improved outcomes for patients with BRAF‐mutated melanomas, their efficacy is often short‐lived, and ...

3. clin.larvol.com clin.larvol.com/trial-detail/NCT07237100

Mirdametinib in Patients With Advanced NF1-mutant ... To evaluate preliminary clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous ...

4. cancernetwork.com cancernetwork.com/view/objective-data-highlight-substantial-benefits-with-mirdametinib-in-nf1-pn

Objective Data Highlight Substantial Benefits With ... Among adult patients with NF1-PN who received mirdametinib, treatment yielded significant improvements in worst tumor pain severity (P <.001), ...

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.24.01034

ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults ... The majority (71%) of adults at cycle 13 reported an improvement (very much/moderately/a little better) in overall status with mirdametinib ( ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11825507/

ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and ... The majority (71%) of adults at cycle 13 reported an improvement (very much/moderately/a little better) in overall status with mirdametinib ( ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2025/219379Orig1s000,219389Orig1s000MultidisciplineR.pdf

219379Orig1s000 219389Orig1s000 - accessdata.fda.gov The pivotal safety data supporting the safety evaluation of mirdametinib is based on Study ... represent possible outcomes for mirdametinib overdoses.

8. targetedonc.com targetedonc.com/view/lifirafenib-plus-mirdametinib-shows-tolerable-safety-in-braf-kras-mutant-advanced-solid-tumors

Lifirafenib Plus Mirdametinib Shows Tolerable Safety in ... These TEAEs lead to dose modification in 57.7%, discontinuation of treatment in 5.6%, and death in 5.6%. Based on these findings, Solomon ...

9. onclive.com onclive.com/view/fda-approves-mirdametinib-for-nf1-associated-plexiform-neurofibromas

FDA Approves Mirdametinib for NF1-Associated Plexiform ... Regarding safety, any-grade treatment-related adverse effects (TRAEs) occurred in 98% of adult patients and 95% of evaluable pediatric patients.