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New treatment option for Langerhans Cell Histiocytosis (LCH)
Official title Mirdametinib in Histiocytic Disorders
ClinicalTrials.gov ID: NCT06153173
What this study is testing
What is Mirdametinib?
Mirdametinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for langerhans cell histiocytosis (lch).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- people must be ≥ 2 years of age AND have a diagnosis of a histiocytic disorder that requires systemic therapy
- 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...
- Must have measurable disease on PET scan or brain MRI
You likely can't join if
- Prior therapy with stipulations as described:
- Myelosuppressive Chemotherapy: Must not have received any cytotoxic chemotherapy which impacts the growth and development of cells in the bone marrow...
- 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...
- Steroids: Due to the increased risk of an ocular event, the use of systemic oral, inhaled, or ocular glucocorticoid therapy is prohibited within 14...
- Radiation: Must not have received radiation within 14 days of study enrollment or have received radiation to the orbit at any time
- Risk factors for retinal vein occlusion (RVO) are listed. Exclusion should be considered by clinical discretion if they have any of the following...
See the full eligibility criteria
- people must be ≥ 2 years of age AND have a diagnosis of a histiocytic disorder that requires systemic therapy
- 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
- Must have measurable disease on PET scan or brain MRI
- people 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
- Prior therapy with stipulations as described:
- 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...
- 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...
- Radiation: Must not have received radiation within 14 days of study enrollment or have received radiation to the orbit at any time
- 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, side effects), ophthalmologist's exam findings and overall assessment will be utilized. If in the ophthalmologist's assessment...
- 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
- LVEF \< 55% at screening OR history of clinically significant cardiac disease, unless deemed to be the direct result of disease
- people 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
The study team makes the final eligibility decision.
Where it's taking place
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Cincinnati, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.