Actively Recruiting
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Led by Johns Hopkins University · Updated on 2026-04-13
24
Participants Needed
1
Research Sites
248 weeks
Total Duration
On this page
Sponsors
J
Johns Hopkins University
Lead Sponsor
S
SpringWorks Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
CONDITIONS
Official Title
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Meet the diagnostic criteria for Neurofibromatosis type 1 (NF1)
- Be 18 years of age or older
- Have at least 24 measurable cutaneous neurofibromas located in two target areas, including at least 6 on the back
- Cutaneous neurofibromas must be non-pedunculated, surrounded by uninvolved skin, and measure at least 0.5 cm in longest diameter and height
- Willing to undergo tumor biopsies before and during treatment if needed
- Karnofsky performance status of 80% or higher
- Adequate organ and bone marrow function based on screening laboratory values
- Able and willing to comply with the study protocol
- Able to understand and willing to sign informed consent
- Women of childbearing potential must not be pregnant or breastfeeding during the study and must use effective contraception during the study and for 6 months after
- Male participants must agree to use contraception or abstain from heterosexual intercourse during the study and for 90 days after last dose
You will not qualify if you...
- Alanine transaminase (ALT) levels more than twice the upper limit of normal
- Total bilirubin greater than 1.5 times the upper limit of normal, unless direct bilirubin is less than 35%
- History of malignancy-associated hypercalcemia
- Active parathyroid disorder or hyperphosphatemia
- Significant liver disease or biliary abnormalities except Gilbert's syndrome or asymptomatic gallstones
- Positive hepatitis B or C tests unless viral load is below defined limits
- History of lymphoma, leukemia, or malignancy in past 5 years except certain skin cancers
- Breast cancer within past 3 years
- Active optic or other low-grade gliomas requiring treatment
- Abnormal corrected QT interval on ECG
- Significant cardiac events or disease within 6 months
- Left ventricular ejection fraction below 55% or history of congestive heart failure
- Retinal conditions or risk factors for retinal vein occlusion
- History of glaucoma
- Positive HIV test
- Gastrointestinal conditions impairing drug absorption
- Prior treatment with MEK inhibitors causing adverse events or within 12 months
- Radiation therapy within 6 months or any radiation to the orbit
- Pregnant or breastfeeding women
- Participation in another clinical study within 28 days
- Known sensitivity to study drug or components
- Use of systemic or ocular glucocorticoids within 14 days before treatment
- Severe uncontrolled medical or social conditions
- Use of pan-cytochrome P450 inducers within 14 days before treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Actively Recruiting
Research Team
C
Carlos Romo, MD
CONTACT
J
Joshua Roberts
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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