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NCT07891286Not yet recruitingPhase 2

Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults

A Phase II Study to Evaluate the Use of the MEK Inhibitor Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults

Source: ClinicalTrials.gov · Last updated 29 Oct 2026

Brief summary (registry, in English)

Background: Large and giant congenital melanocytic nevi (LGCMN) are dark birthmarks (moles). They can grow to 8 inches or more across. People with LGCMN have an increased risk of getting skin cancer or problems with the nervous system. Researchers want to find out if a drug approved for a different use (mirdametinib) can shrink the size of LGCMN. Objective: To test mirdametinib in people with LGCMN. Eligibility: People aged 3 to 40 years with LGCMN. Design: Participants will be screened. Their LGCMN sites will be measured and photographed. They will have blood tests, an eye exam, and tests of their heart function. They will also have: * An exam of their nervous system: Their muscle strength, ability to feel and touch, balance, alertness, and other skills will be tested. * Magnetic resonance imaging (MRI) scan: They will lie on a bed that slides into a tube; the scan will use large magnets to create images of their brain and spinal cord. * Skin biopsy: A small sample of their skin may be taken. * VECTRA scan: This is a 3-dimensional imaging system that takes pictures of all of the skin all at once. Mirdametinib will be either pills or capsules that are taken by mouth. Participants will take the drug in 4-week cycles. They will take the pills 2 times a day for 3 weeks with 1 week off. Participants may continue taking the drug for 2 years. They will have clinic visits 12 times while taking the drug. Imaging scans and other tests will be repeated. Participants will continue to have checkups every 6 months for 3 more years.

Status (registry)
Not yet recruiting
Phase (registry)
Phase 2
Study type
Interventional
Enrollment
15 (estimated)
First submitted
22 Oct 2026
Start date
4 Jan 2027
Primary completion date
4 Jan 2029
Lead sponsor
National Cancer Institute (NCI)
NCT number
NCT07891286

What you commit to

  • Study type: Interventional · Phase 2
  • Planned participants: 15
  • From 4 Jan 2027 to the primary completion date, 4 Jan 2029
  • 1 site in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Participants who are receiving any investigational agents within the 30 days prior to the study treatment initiation.
  • Prior treatment with mirdametinib or another specific MEK1/2 inhibitor.
  • Cardiac conditions:
    • Heart failure (any New York Heart Association \[NYHA\] class)
    • Myocardial infarction, severe/unstable angina, coronary or peripheral artery bypass graft placement, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism within 6 months prior to starting study treatment.
  • Any unresolved chronic toxicity grade \>= 2 per CTCAE from previous therapy, except for alopecia.
  • Major surgery within 4 weeks prior to starting study treatment.
  • Prior history of melanoma or other malignancy.
  • Active infection (currently on treatment with anti-microbials).

Other illness

  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to mirdametinib.
  • Refractory nausea and vomiting, chronic gastrointestinal disease (e.g., inflammatory bowel disease), or significant bowel resection that would preclude adequate absorption of mirdametinib.
  • History of renal transplant.
  • Uncontrolled, symptomatic intercurrent illness evaluated by medical history, physical exam, lab evaluations that would unacceptably increase risk for the participant or impair the ability to evaluate the endpoints of the study.

Lab values

  • Ophthalmologic conditions:
    • Current or history of central serous retinopathy
    • Current or history of retinal vein occlusion
    • Known intraocular pressure (IOP) \> 21 mmHg (or ULN adjusted by age) or uncontrolled glaucoma (irrespective of IOP).
    • Ophthalmologic findings secondary to long-standing optic pathway glioma or NCM (such as visual loss, optic nerve pallor, or strabismus) will NOT be considered a significant abnormality for the purposes of the study.
  • Positive serum or urine beta-human chorionic gonadotropin (beta-HCG) pregnancy test performed at screening.

Criteria the registry did not label

  • INCLUSION CRITERIA:
  • Participants must have a clinical diagnosis of a large and giant congenital melanocytic nevi (LGCMN).
  • Participants must have a documented history of somatic pathogenic or likely pathogenic variation in NRAS or BRAF, or a pathogenic fusion in which one of the fusion partners is ALK, NTRK1, BRAF, or RAF1. Participants who do not have documented history will be tested by NSR device (via next-generation sequencing panel TruSight(TM) Oncology 500)
  • Participants must have: at least one nevus \> 20 cm
    • OR
    • at least one nevus with a projected adult diameter \> 20 cm (participants \< 18 years old).
    • Note: Participants who have had prior surgical treatment of their LGCMN, but the remaining nevus has a diameter \> 20 cm, are eligible for enrollment.
  • Age \>= 3 years and \<= 40 years old.
  • Participants must have a Body Surface Area (BSA) \>= 0.4 m\^2.
  • Clinical performance status: Karnofsky \>= 70% (\>= 16 years); Lansky scale \>=70% (\< 16 years).
  • Participants must have adequate organ and marrow function as defined below:
    • Hemoglobin \>= 11 g/dL
    • Absolute neutrophil count \>= 1,000/mcL
    • Platelets \>= 100,000/mcL
    • Total bilirubin \<= 1.5 X institutional upper limit of normal (ULN) for age. Note: 3 X ULN for age for participants with Gilbert syndrome
    • Aspartate Aminotransferase (AST) \<= 3 X institutional ULN
    • Alanine Aminotransferase (ALT) \<= 3 X institutional ULN
    • Estimated creatinine clearance \>= 30 mL/min/1.73 m\^2 as calculated per the 2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (\>= 18 years old) or Chronic Kidney Disease in Children Under 25 (CKiD U25) in (\< 18 years old)
    • Cardiac function: Normal ejection fraction (echocardiogram \[ECHO\] or cardiac MRI), defined as ejection fraction (EF) \>= 53%; normal QTc interval, defined as QTcF \<= 450 msec
    • Blood pressure (BP):
    • Adults (\>= 18 years old): BP \<= 130 systolic and \<= 80 diastolic
    • Children (\< 18 years old): BP \<= the 95th percentile for age, height, and sex
    • Adequate blood pressure can be achieved using medication for the treatment of hypertension.
    • --Serum creatine phosphokinase (CPK) \<= 2.5 X institutional ULN
  • Participants with neurocutaneous melanocytosis must be asymptomatic and have nonprogressive disease.
  • Participants with seizure disorder may be enrolled if on anticonvulsants, and there has been no change to their anticonvulsant medication in the 4 weeks prior to the study treatment initiation, and the participant has been seizure-free during that time.
  • Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy must have an undetectable viral load.
  • Participants with evidence of chronic hepatitis B virus (HBV) infection must have an undetectable HBV viral load and be on suppressive therapy, if indicated.
  • Participants with evidence of hepatitis C virus (HCV) infection must have an undetectable HCV viral load and be cured or on suppressive therapy, if indicated.
  • Women who are nursing or plan to nurse a child must agree to discontinue/postpone nursing while taking mirdametinib and until 1 week after the administration of the last dose of mirdametinib.
  • Women of child-bearing potential (WOCBP) must agree to use an effective contraception (barrier, hormonal, intrauterine device, surgical sterilization, continuous abstinence) while taking mirdametinib and until 6 weeks after administration of the last dose of mirdametinib. A participant may request a male partner to use an effective form of contraception to fulfill this requirement.
    • Note: WOCBP defined as:
  • biologically female sex with a uterus and at least one ovary who is at least Tanner Stage 3, AND
  • entered puberty and has not completed menopause AND
  • does not meet the definition of female participant who cannot get pregnant as follows:
    • has undergone a hysterectomy OR
    • has undergone a bilateral oophorectomy OR
    • has completed menopause defined as 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels \> 40 mIU/ml or 6 weeks postsurgical OR
    • has not gone through menarche or reached Tanner Stage 3
    • Men (biologically male sex) must agree to use an effective method of contraception (barrier, surgical sterilization, abstinence) while taking mirdametinib and until 3 months after administration of the last dose of mirdametinib. A participant may request a female partner to use an effective form of contraception to fulfill this requirement. Men must not freeze or donate sperm within the same period.
    • -Ability of participant or parent/guardian to understand and the willingness to sign a written informed consent document.

Age in the registry: 3 years to 40 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
National Institutes of Health Clinical Center
Coordinator of this site? Confirm its status		BethesdaUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.