Trials / Recruiting
RecruitingNCT06153173
Mirdametinib in Histiocytic Disorders
A Phase II Trial of the MEK Inhibitor Mirdametinib in Histiocytic Disorders
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- Sponsor
- Children's Hospital Medical Center, Cincinnati · Academic / Other
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Not accepted
Summary
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.
Detailed description
Langerhans cell histiocytosis (LCH) is a rare blood disorder. Though affecting all ages, LCH occurs more often in children, with an increased incidence in children less than 1 year of age. The disease presents in various ways, with most children suffering bony lesions, and skin rashes. In some patients, LCH affects vital organs such as liver, spleen, bone marrow, and the central nervous system. This group of patients are at significant risk of serious illness and death and are thus said to have risk-organ-positive (RO+) LCH. Current treatments for LCH consist of chemotherapy combined with other medications. However, many patients, especially those with RO+ disease, do not respond to therapy. Of the patients that do respond, many suffer progression of disease after an initial response to therapy, or recurrence of disease after completion of therapy. The purpose of this study is to see if treatment with mirdametinib in patients with LCH or other histiocytic disorders will be better than current treatments and with fewer side effects.
Conditions
- Langerhans Cell Histiocytosis (LCH)
- Juvenile Xanthogranuloma (JXG)
- Rosai-Dorfman Disease (RDD)
- Histiocytic Disorders
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Mirdametinib | Mirdametinib is administered by mouth twice daily on a continuous schedule, with each cycle being 4 weeks. Patients are instructed to take consecutive doses separated by a minimum of 6 hours and a maximum of 14 hours. |
Timeline
- Start date
- 2024-02-05
- Primary completion
- 2030-03-01
- Completion
- 2031-03-01
- First posted
- 2023-12-01
- Last updated
- 2025-09-16
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06153173. Inclusion in this directory is not an endorsement.