Back to search results

Mirdametinib Monotherapy for Adults with NF1 and Cutaneous Neurofibromas

This study is testing a drug called mirdametinib for adults who have Neurofibromatosis 1 (NF1) and cutaneous neurofibromas (cNFs), which are skin tumors. The main goals are to find a safe dose of mirdametinib and see if it helps shrink these tumors. Researchers will look at different ways to give the drug, either continuously or with breaks. To join, you need to be at least 18 years old and have at least 24 measurable cNFs that meet specific size and location requirements. The study is currently recruiting about 24 participants.

Study design
This is a Phase 1/2a, open-label (meaning you and the researchers will know what treatment you are receiving), non-randomized study. It will involve about 24 participants.
What's involved
Participation involves a screening period, a treatment period that could last up to 48 cycles (each cycle is 28 days), and a safety follow-up period after treatment ends. All study activities will take place at Johns Hopkins University.
Compensation
Not stated in the trial record.
Follow-up
There will be a post-study safety follow-up period after treatment ends, but the duration is not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06159166

Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).

Recruiting
PHASE1Ages 18+InterventionalTreatment
Johns Hopkins University
~24 participants
Updated 2026-04-13 on ClinicalTrials.gov
What's tested:Mirdametinib

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Assess the safety of continuous and three week on-one week off dosing of mirdametinib in adults with NF1 and cutaneous neurofibromas (cNFs).
Measured over Up to 28 days
+2 more outcomes measured

Conditions

NF1
Cutaneous Neurofibroma
Monotherapy

Explore related trials

Neurofibromatosis Type 1Skin Neurofibroma

Where it's being run

1 sites across 1 states
Maryland1

Study leadership

  • Carlos Romo, MD · PRINCIPAL_INVESTIGATOR · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Who to contact

Carlos Romo, MD
[email protected]
(410) 955-6827
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

In order for a woman to be determined not of childbearing potential, she must have ≥ 12 months of non-therapy-induced amenorrhea or be surgically or medically sterile.
WOCBP must have a negative serum pregnancy test result at Screening and a negative urine pregnancy test result at the Baseline visit prior to the first dose of study treatment.
The Investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy. 10. Male participants are eligible to participate if they agree to the following during the treatment period and for at least 90 days after the last dose of study treatment:
Refrain from donating sperm
Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent until 90 days after the last study drug treatment; OR
Must agree to use a male condom when having sexual intercourse with a WOCBP and their female partner must utilize one the approved methods of birth control below:
Total abstinence
Male or female sterilization (vasectomy in males or surgical removal of ovaries or uterus in females)
Unsterilized male study participants must use a male condom and their female partner must use one of the methods below:
Unsterilized female study participants must use one of the following highly effective methods listed below:
Combined (estrogen and progestogen containing) hormonal contraceptive that stops the release of eggs from the ovary (oral, intravaginal, or transdermal)
Progestogen-only hormonal contraception that stops the release of eggs from the ovary (oral, injectable, implantable)
Intrauterine device (IUD)
Intrauterine hormone-releasing system (IUS)
Bilateral tubal occlusion or bilateral tubal ligation

Exclusion

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);
Hypertension (BP ≥ 140/90 mm Hg) 13. History of glaucoma; 14. Known history of a positive human immunodeficiency virus (HIV) antibody test; 15. Known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of mirdametinib (e.g., gastric bypass, lap band, or other gastric procedures). Delivery of mirdametinib via nasogastric tube or gastrostomy tube is not allowed. 16. Previously treated with MEK inhibitor including mirdametinib (PD-0325901) and had to stop treatment due to adverse event. 17. Currently receiving therapy with a MEK inhibitor including mirdametinib (PD-0325901) or treated with a MEK inhibitor in the 12 months prior to prior to first dose of study treatment. 18. Received radiation therapy within the 6 months prior to prior to first dose of study treatment. Participants who have received radiation to the orbit at any time are excluded. 19. Pregnant or breastfeeding women may not take study drug. 20. Current enrollment or past participation in any other clinical study (excluding observational studies) within 28 days of first dose of study treatment. 21. Known sensitivity to the study treatment, or components thereof, or drug or other allergy that, could compromise safety of the subject 22. Participant is receiving systemic (oral, inhaled, of IV/SC) or ocular glucocorticoid therapy (with the exception of participants with endocrine deficiencies who are allowed to receive physiologic or stress doses of steroids, if necessary) within 14 days prior to first dose of study treatment; 23. Participants are excluded if they have severe and/or uncontrolled medical disease or social situation, which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration, congestive heart failure, drug or alcohol dependence, etc.). 24. Participants is receiving systemic treatment of a pan-cytochrom 450 (CYP) inducers such as rifampin or ritonavir within 14-days prior to first dose of study treatment Drug Development and Drug Interactions

What this trial measures

  • Assess the safety of continuous and three week on-one week off dosing of mirdametinib in adults with NF1 and cutaneous neurofibromas (cNFs).Up to 28 days

    Phase 1 will test the safety and tolerability of multiple dose regimens of mirdametinib, in order to identify the recommended phase 2 dose(s) for phase 2a. The study will continue until one of the stopping conditions is met. * The RP2D(s) have been identified with sufficient accuracy (The dose regimen(s) selected as the RP2D have been fully enrolled with at least 3 cycles of treatment completed for all participants) * All dose regimens are deemed to have unacceptable safety The study will continue until one of the following stopping conditions is met. * The RP2D(s) have been identified with sufficient accuracy (The dose regimen(s) selected as the RP2D have been fully enrolled with at least 3 cycles of treatment completed for all participants) * All dose regimens are deemed to have unacceptable safety

  • Define the recommended phase 2 dose (RP2D) of mirdametinib in adults with NF1 and cNF.Up to 28 days

    Phase 2a of the study will test the efficacy and safety of a maximum of two RP2Ds in adults with NF1 and cNF.

  • • Evaluate the preliminary anti-tumor activity of mirdametinib monotherapy in adults with NF1 and cNFs.Up to 28 days

    Evaluate the efficacy of mirdametinib monotherapy at the RP2D in adults with NF1 and cNF. We will determine efficacy of Anti-tumor activity with 3D photography (Sum of the surface area, sum of the tumor volume, sum of the longest diameter) and Digital Caliper (Sum of the longest diameter).