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Mirdametinib for Histiocytic Disorders

This study is testing a medication called mirdametinib for people with histiocytic disorders, including Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG), and Rosai-Dorfman Disease (RDD). Mirdametinib is taken by mouth twice daily. The goal is to see if mirdametinib works better and has fewer side effects than current treatments. The study is looking for people aged 2 years and older who have a histiocytic disorder that needs systemic (whole-body) treatment. Researchers will measure how well the treatment works after one year. The study is currently recruiting 40 participants.

Study design
This study plans to enroll 40 participants. It is an interventional study, meaning participants will receive a specific treatment.
What's involved
Participants will take mirdametinib by mouth twice daily, with doses separated by at least 6 hours and no more than 14 hours. Each treatment cycle lasts 4 weeks.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint, response rate to mirdametinib, is measured at 1 year (completion of 13 four-week cycles).

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

NCT06153173

Mirdametinib in Histiocytic Disorders

Recruiting
PHASE2Ages 2+InterventionalTreatment
Children's Hospital Medical Center, Cincinnati
~40 participants
Updated 2025-09-16 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
Response rate to mirdametinib
Measured over 1 year (completion of 13 four week cycles)

Conditions

Langerhans Cell Histiocytosis (LCH)
Juvenile Xanthogranuloma (JXG)
Rosai-Dorfman Disease (RDD)
Histiocytic Disorders

Explore related trials

Histiocytic DisorderJuvenile XanthogranulomaLangerhans Cell HistiocytosisRosai-Dorfman-Destombes DiseaseHistiocytic and Dendritic Cell Neoplasm

NCT06153173

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohiostudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Ashish Kumar, MD, PhD · PRINCIPAL_INVESTIGATOR · Children's Hospital Medical Center, Cincinnati
  • Allison Bartlett, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital Medical Center, Cincinnati

Who to contact

Monica Trapp
[email protected]
(513) 803-8574
Email the study team

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Eligibility criteria

Inclusion

If patient has had a diagnostic biopsy, biopsy must be reviewed and confirmed by CCHMC pathologist as feasible
If patient has had a biopsy but has not had molecular testing done, must have tissue available for mutational analysis
If patient has isolated pituitary/CNS disease or situations where biopsy is not feasible, positive ddPCR blood test for mutation associated with histiocytic neoplasm with clinical features of histiocytosis is sufficient 2. Must have measurable disease on PET scan or brain MRI 3. Subjects must demonstrate adequate organ function as defined:
Renal: maximum serum creatinine 2x the upper limit of normal (ULN) OR a creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2
Liver: ALT ≤ 3x ULN AND normal INR (≤ 1.5)
Hematologic: Hematology: Albumin ≥ 2.8 g/dL; Absolute neutrophil count ≥ 1.5 x 109/L; Platelets ≥ 100 x 109/L; Hemoglobin ≥ 9.0 g/dL
Patients with organ function abnormalities outside of these thresholds deemed to be the result of histiocytic disease will be considered eligible

Exclusion

Myelosuppressive Chemotherapy: Must not have received any cytotoxic chemotherapy which impacts the growth and development of cells in the bone marrow within 14 days of enrollment onto this study (i.e. cytarabine, cladribine, clofarabine, mercaptopurine, methotrexate, vinblastine)
MEK Inhibitors: Must not have received a MEK inhibitor within 30 days (or 5 half-lives, whichever is longer) of enrollment, NOR have had disease progression on MEK inhibitor
Steroids: Due to the increased risk of an ocular event, the use of systemic oral, inhaled, or ocular glucocorticoid therapy is prohibited within 14 days prior to first dose of mirdametinib. Throughout the treatment period, short term glucocorticoid treatment (30 days or less) is permitted. Any patients requiring long-term steroid use (more than 30 consecutive days) are not eligible. The exception to this rule is subjects with endocrine deficiencies who require physiologic steroids
Radiation: Must not have received radiation within 14 days of study enrollment or have received radiation to the orbit at any time 2. Risk factors for retinal vein occlusion (RVO) are listed. Exclusion should be considered by clinical discretion if they have any of the following risk factors for RVO at screening:
Intraocular pressure (IOP) \> 21 mmHg; if IOP is unable to be obtained (eg age, cooperation, tolerability), ophthalmologist's exam findings and overall assessment will be utilized. If in the ophthalmologist's assessment there are no signs of raised IOP, the subject will be considered eligible for this parameter
Glaucoma or any significant abnormality (≥ grade 2) on ophthalmologic exam that is uncontrolled with intervention
Serum cholesterol \> 300 mg/dL
Serum triglycerides \> 300 mg/dL
Hyperglycemia (either fasting blood glucose \> 125 mg/dL OR random blood glucose \> 200 mg/dL)
Uncontrolled hypertension (participants ≤ 12 years of age with a blood pressure ≥ 95th percentile for age + 12 mmHg; participants ≥ 13 years of age with a blood pressure ≥ 140/90 mm Hg) unresolved on repeat measurement 3. LVEF \< 55% at screening OR history of clinically significant cardiac disease, unless deemed to be the direct result of disease 4. Subjects who are pregnant or breastfeeding, or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study

What this trial measures

  • Response rate to mirdametinib1 year (completion of 13 four week cycles)

    Best overall response rate to mirdametinib after 13 four-week cycles as defined by positron emission tomography (PET) or magnetic resonance imaging (MRI) (for isolated pituitary/central nervous system (CNS) disease) response criteria.