Actively Recruiting

Phase 2
Age: 2Years +
All Genders
NCT06153173

Mirdametinib in Histiocytic Disorders

Led by Children's Hospital Medical Center, Cincinnati · Updated on 2025-09-16

40

Participants Needed

1

Research Sites

368 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

The purpose of this study is to see if treatment with mirdametinib in patients with Langerhans cell histiocytosis (LCH) or other histiocytic disorders will be better than current treatments and with fewer side effects.

CONDITIONS

Official Title

Mirdametinib in Histiocytic Disorders

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must be 2 years of age or older with a diagnosis of a histiocytic disorder requiring systemic therapy
  • Diagnostic biopsy must be reviewed and confirmed by pathologist if available
  • Tissue must be available for mutation testing if biopsy performed without prior molecular analysis
  • Positive blood mutation test plus clinical features accepted if biopsy not feasible
  • Measurable disease must be present on PET scan or brain MRI
  • Adequate organ function including kidney, liver, and blood counts as defined by study thresholds
  • Organ function abnormalities caused by histiocytic disease may be considered eligible
Not Eligible

You will not qualify if you...

  • Received cytotoxic chemotherapy affecting bone marrow within 14 days prior to enrollment
  • Received MEK inhibitors or had disease progression on MEK inhibitors within 30 days prior to enrollment
  • Use of systemic, inhaled, or ocular steroids within 14 days before first dose, except short-term use or physiologic steroids for endocrine deficiencies
  • Received radiation therapy within 14 days prior to enrollment or to the orbit at any time
  • Risk factors for retinal vein occlusion including high eye pressure, glaucoma, high cholesterol, triglycerides, hyperglycemia, or uncontrolled hypertension
  • Left ventricular ejection fraction less than 55% or clinically significant heart disease unless caused by disease
  • Pregnant or breastfeeding or unable to use acceptable birth control methods during the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

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Research Team

M

Monica Trapp

CONTACT

C

Caitlin Cottrell

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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