Advanced Unresectable MelanomaPhase 2NCT07237100
Mirdametinib in Patients With Advanced NF1-mutant Melanoma
A Phase 2 study testing Mirdametinib in people with Advanced Unresectable Melanoma and Metastatic Melanoma, run by Kevin Kim, MD at 1 site in United States.
Trial summary
The goal of this clinical trial is to evaluate clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Unresectable Melanoma, Metastatic Melanoma
- What is being tested?
- Mirdametinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Oct 2025to 13 Oct 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with unresectable or metastatic melanoma with an NF1 mutation; Variance of NF1 of unknown/ uncertain significance will not be eligible; The genetic analysis for somatic mutations must be performed in a lab that has obtained CLIA certification.
- Patients must have a report of NF1 sequencing analysis performed at CLIA-certified laboratory (by either tissue-based sequencing or liquid biopsy)
- Must have been previously treated with
- anti PD-1/PD-L1 antibody; AND anti CTLA-4 antibody and/or anti LAG3 antibody;
- UNLESS these standard checkpoint inhibitors are not clinically indicated or suitable (for example, comorbid conditions, such as autoimmune disease, or significant toxicity with prior checkpoint inhibitor treatment)
- Tumors must be progressing at the time of the enrollment
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- Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
- Patients must be ≥ 18 years of age
- Patients must have measurable metastatic disease according to RECIST 1.1
- Patients must have adequate organ function, defined as follows:
- Absolute neutrophil count ≥ 1,500/μL
- Platelets ≥ 100,000/μL
- Hemoglobin ≥ 9 g/dL
- Serum creatinine ≤ 1.5 x upper limit of normal (ULN) or calculated creatinine clearance ≥ 30 mL/min using the Cockcroft-Gault equation. Estimated glomerular filtration rate (eGFR) ≥60 mL/min/1.73 m2 calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Grade ≤1). • Total bilirubin ≤ 1.5 x ULN OR direct bilirubin ≤ 1 x ULN (except patients with Gilbert's Syndrome, who must have a total bilirubin less than 3.0 mg/dL)
- Aspartate aminotransferase and alanine aminotransferase ≤ 3.0 x ULN (Grade ≤1) unless liver metastases are present, in which case they must be ≤ 5 x ULN (Grade ≤2).
- Adequate coagulation function, as determined by:
- International Normalized Ratio (INR) ≤ 1.5 × ULN (Grade ≤ 1). If the participant receives anticoagulant therapy, the INR > 1.5 × ULN is permitted, but the dose must be stable for at least 2 weeks before the start of the study treatments.
- PTT ≤ 1.5 × ULN.
- Adequate cardiac function, as determined by:
- Systolic blood pressure < 160 mmHg and diastolic blood pressure < 100 mmHg (Grade ≤ 2).
- LVEF ≥ 50% by MUGA or ECHO.
- No clinically significant ECG waveform abnormalities assessments at screening (one triplicate).
- Have normal serum calcium and phosphate levels (calcium level may be corrected for albumin level).
- Female patients are eligible to enroll and participate in the study if:
- Patient is of non-childbearing potential (i.e., physiologically incapable of becoming pregnant), including any female who:
- has had a hysterectomy.
- has had a bilateral oophorectomy (ovariectomy).
- has had bilateral tubal ligation.
- is postmenopausal (total cessation of menses for ≥1 year), OR
- Women of child-bearing potential must agree to use highly effective contraceptive methods (hormonal or barrier method of birth control or abstinence) during the trial period through at least six months after the last dose. Male patients or their partners must be surgically sterile or agree to use adequate contraception while receiving trial treatment and for three months thereafter. Contraceptive use by men or women should be consistent with Clinical Trials Coordination Group (CTCG) guidance.
- Patients must be able to understand the study procedures and agree to participate in the study by providing written informed consent.
Who cannot join
- Patients who were previously treated with MEK, ERK or RAF inhibitor therapy
- Patients with symptomatic brain metastasis or active brain lesions ≥ 6 mm size or those who require steroid treatment for brain lesions or leptomeningeal disease
- No systemic cancer therapy within 28 days of the study drug administration,
- Patients must not be simultaneously enrolled in any therapeutic clinical trial
- Patients must not have had investigational therapy administered ≤ 4 weeks, or within a time interval less than at least 5 half-lives of the investigational agent, whichever is longer, prior to the first scheduled day of dosing in this study.
- Patient has a history of, or evidence of, retinal pathology on ophthalmologic examination that is considered a risk factor for central serous retinopathy, retinal vein occlusion (RVO), or neovascular macular degeneration. Participants will be excluded from study participation if they have any of the following risk factors for RVO at Screening:
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- Intraocular pressure > 21 mmHg;
- Serum cholesterol > 300 mg/dL;
- Serum triglycerides > 300 mg/dL;
- Hyperglycemia (fasting blood glucose > 125 mg/dL or random blood glucose > 200 mg/dL);
- History or current evidence of glaucoma or clinically significant abnormalities on the ophthalmological exam, including but not limited to cataract limiting the ability to examine the retina or any optical coherence tomography (OCT) finding that could be a significant risk factor for RVO, retinopathy or neovascular macular degeneration.
- o Note: Mild and controlled/stable age-related macular degeneration or non-proliferative diabetic retinopathy may be acceptable at the investigator's discretion after consultation with the ophthalmologist.
- History (within 6 months before the start of the study treatments) of clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, clinically significant transient ischemic attack, symptomatic pulmonary embolism, unexplained syncope, or long QT syndrome.
- Patient who is pregnant or breastfeeding.
- Patient with active bacterial, fungal, or viral infection, including but not limited to the use of antibiotics, antifungals, or antiviral agents at the time of Screening;
- Underlying medical conditions, laboratory abnormality, or alcohol or drug abuse or dependence that, in the Investigator's opinion, will be unfavorable for the administration of study treatment or affect the explanation of drug toxicity or adverse events; or insufficient compliance during the study according to Investigator's judgement; or
- Patient has experienced other severe acute or chronic medical or psychiatric conditions, including recent (within 1 year of signing informed consent/assent) or active suicidal ideation or behavior, or a laboratory abnormality that may increase the risk associated with study participation or study treatment administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the participant inappropriate for entry into this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Andrea Davila contact
United States1 site, 1 recruiting
- Recruiting
California Pacific Medical Center - Sutter Health
San Francisco, California, 94115
California Pacific Medical Center Research Institute, clinicalresearch@sutterhealth.org
Andrea Davila, andrea.davila@sutterhealth.org
About this study
Official title: A Pilot Study of Mirdametinib in Patients With Advanced Melanoma With an NF1 Mutation
The goal of this clinical trial is to evaluate clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy. The main questions this study aims to answer are:
- To evaluate the feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy.
- To evaluate preliminary clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy
- To evaluate the safety profile of mirdametinib in patients with advanced NF1-mutant melanoma
Read the detailed description
The purpose of this study is to test if mirdametinib is safe and effective in improving disease status and/or delaying progression of disease. Mirdametinib is being studied to see if it can help slow down or stop the growth of certain types of cancer.
Mirdametinib is a "MEK inhibitor," which targets specific pathways inside cancer cells. In some cancers, these pathways are overly active, causing the cells to grow and divide quickly. Mirdametinib is designed to block this activity, potentially slowing or stopping cancer growth.
Because mirdametinib is a targeted therapy, it focuses on the cancer cells more directly rather than attacking healthy cells throughout the body. This means it may work differently from traditional chemotherapy.
- Being tested
- Mirdametinib
- Keywords
- Neurofibromin 1 (NF1) mutation, advanced unresectable melanoma, Metastatic Melanoma, Melanoma, Mirdametinib
- Medical subject headings
- Melanoma, Neurofibromatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MirdametinibDrug
Also called PD-0325901
Mirdametinib is a kinase inhibitor. It is taken by mouth twice a day
| Study group | Role | What they receive |
|---|---|---|
| Mirdametinib | Experimental | 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. Receives: Drug: Mirdametinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy.
This is a small pilot study to evaluate a feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy. The primary endpoint is to enroll and treat 10 patients over a 12 month period. If there are at least 2 clinical responses among the 10 patients, the preliminary efficacy will be considered promising. Further discussions with the funding agency to expand the cohort to a total of 29 patients will then be planned.
Measured: 12 months
Secondary outcomes (2)
To evaluate the safety profile of mirdametinib in patients with advanced NF1-mutant melanoma
Descriptive statistics for safety will be presented using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. All on-study AEs, Grade 3-4 AEs, treatment-related AEs, Grade 3-4 treatment-related AEs, SAEs, AESIs, treatment-related SAEs, and AEs leading to dose reduction, dose interruption, and treatment discontinuation will be tabulated using worst grade per NCI CTCAE v 5.0 criteria by system organ class and preferred term. On-study lab and Grade 3-4 Lab Abnormalities will be summarized using baseline and worst grade NCI CTCAE v 5.0 criteria.
Measured: 2 years
To evaluate preliminary clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy
Descriptive statistics of enrollment rate, overall response rate, progression-free survival and overall survival will be performed. Endpoints for efficacy will include response rate by RECIST 1.1, progression-free survival, and survival by Kaplan-Meier analysis.
If there are at least 2 clinical responses among the 10 patients, the preliminary efficacy will be considered promising.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2025
- Study start13 Oct 2025
- Primary completion13 Oct 2027 (estimated)
- Study completion13 Oct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Unresectable MelanomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The dosing schedule: • 4 mg BID for administered continuously (1 cycle = 28 days).
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kevin Kim, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Kevin Kim, MD, Sutter Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07237100
- Sponsor's protocol ID
- CPMC24-MEL01
- Status last verified
- Nov 2025
- First submitted
- 13 Nov 2025
- Last updated
- 19 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Mirdametinib in Patients With Advanced Melanoma With an NF1 Mutation
- ClinicalTrials.gov ID
- NCT07237100
- Acronym / sponsor ID
- CPMC24-MEL01
- Keywords
- Neurofibromin 1 (NF1) mutation, advanced unresectable melanoma, Metastatic Melanoma, Melanoma, Mirdametinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Kevin Kim, MDUniversity, hospital or other
- Study start
- 13 Oct 2025 (actual)
- Primary completion
- 13 Oct 2027 (estimated)
- Study completion
- 13 Oct 2030 (estimated)
- First posted
- 19 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy. The main questions this study aims to answer are:
- To evaluate the feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy.
- To evaluate preliminary clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy
- To evaluate the safety profile of mirdametinib in patients with advanced NF1-mutant melanoma
Detailed description
The purpose of this study is to test if mirdametinib is safe and effective in improving disease status and/or delaying progression of disease. Mirdametinib is being studied to see if it can help slow down or stop the growth of certain types of cancer.
Mirdametinib is a "MEK inhibitor," which targets specific pathways inside cancer cells. In some cancers, these pathways are overly active, causing the cells to grow and divide quickly. Mirdametinib is designed to block this activity, potentially slowing or stopping cancer growth.
Because mirdametinib is a targeted therapy, it focuses on the cancer cells more directly rather than attacking healthy cells throughout the body. This means it may work differently from traditional chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The dosing schedule: • 4 mg BID for administered continuously (1 cycle = 28 days).
| Arm | Type | Receives |
|---|---|---|
| Mirdametinib | Experimental | Drug: MirdametinibPatients 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 may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (31 listed)
- Patients with unresectable or metastatic melanoma with an NF1 mutation; Variance of NF1 of unknown/ uncertain significance will not be eligible; The genetic analysis for somatic mutations must be performed in a lab that has obtained CLIA certification.
- Patients must have a report of NF1 sequencing analysis performed at CLIA-certified laboratory (by either tissue-based sequencing or liquid biopsy)
- Must have been previously treated with
- Tumors must be progressing at the time of the enrollment
- Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
Key exclusion criteria (17 listed)
- Patients who were previously treated with MEK, ERK or RAF inhibitor therapy
- Patients with symptomatic brain metastasis or active brain lesions ≥ 6 mm size or those who require steroid treatment for brain lesions or leptomeningeal disease
- No systemic cancer therapy within 28 days of the study drug administration,
- Patients must not be simultaneously enrolled in any therapeutic clinical trial
- Patients must not have had investigational therapy administered ≤ 4 weeks, or within a time interval less than at least 5 half-lives of the investigational agent, whichever is longer, prior to the first scheduled day of dosing in this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MirdametinibAlso: PD-0325901Mirdametinib is a kinase inhibitor. It is taken by mouth twice a day | Drug | Mirdametinib |
Primary outcome
To evaluate the feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy.
Time frame: 12 months
This is a small pilot study to evaluate a feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy. The primary endpoint is to enroll and treat 10 patients over a 12 month period. If there are at least 2 clinical responses among the 10 patients, the preliminary efficacy will be considered promising. Further discussions with the funding agency to expand the cohort to a total of 29 patients will then be planned.
2 secondary outcomes
To evaluate the safety profile of mirdametinib in patients with advanced NF1-mutant melanoma
Time frame: 2 years
To evaluate preliminary clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2025.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andrea Davila contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mirdametinib in people with Advanced Unresectable Melanoma and Metastatic Melanoma, run by Kevin Kim, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 31 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Research at Sutter Health (415-600-1544, clinicalresearch@sutterhealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the feasibility of conducting a prospective single-center clinical trial of mirdametinib in patients with advanced NF1-mutant melanoma whose disease progresses during or after PD-1 antibody-based checkpoint inhibitor therapy. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2025.