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New treatment option for Langerhans Cell Histiocytosis
Official title Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib
ClinicalTrials.gov ID: NCT06582745
What this study is testing
What is Trametinib?
Trametinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for langerhans cell histiocytosis.
Also referred to as Mekinist.
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 Phase II clinical trial is to establish the safety and effectiveness of trametinib, a targeted therapy, for the treatment of newly or recently diagnosed Langerhans Cell Histiocytosis (LCH) among pediatric patients.
- 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 1 to 30
You may be able to join if
- Diagnosis/disease status:
- Patients with newly diagnosed Langerhans cell histiocytosis (LCH) OR
- Patients with relapsed or refractory disease OR
- Patients with newly diagnosed or relapsed/refractory disease who are receiving the liquid formula of trametinib OR
- Patients who have been receiving trametinib as a treatment for LCH since January 1, 2020 may be included in the observational chart review to track...
You likely can't join if
- Patients diagnosed with Low-Risk True Skin Only or a Single Bone lesion that does not require treatment and will only be observed will not be...
- CNS-risk/special site includes: Sphenoid, Mastoid, Orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or...
- Functionally critical: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local...
- Patients whose genetic testing reveals a class 3 MAP2K1 mutation:
- I103\_K104del
- E102\_I103del
See the full eligibility criteria
- Diagnosis/disease status:
- Patients with newly diagnosed Langerhans cell histiocytosis (LCH) OR
- Patients with relapsed or refractory disease OR
- Patients with newly diagnosed or relapsed/refractory disease who are receiving the liquid formula of trametinib OR
- Patients who have been receiving trametinib as a treatment for LCH since January 1, 2020 may be included in the observational chart review to track long-term follow-up. Eligibility for chart review cohort will include...
- Diagnosis confirmed with biopsy prior to start of treatment
- Patient must have adequate cardiac function evident through Echocardiogram (ECHO) and Electrocardiogram (EKG) within 30 days of starting treatment.
- Shortening fraction of ≥ 27% by echocardiogram or
- Ejection fraction of ≥ 50% by gated radionuclide study
- QTC \< 480 msec
- Performance status: Patients must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥50% for patients ≤16 years of age.
- Adequate organ and marrow function as defined below:
- Absolute Neutrophil count ≥ 1,500/μL
- Platelets ≥ 100x103/μL
- Total bilirubin ≤ 1.5X ULN for age
- AST/ALT ≤ 2.5 X ULN for age
- Serum creatinine based on age/gender
- Hemoglobin ≥ 8 g/dL
- Patients with bone marrow disease must have hemoglobin ≥ 8 g/dL with transfusion support allowed
- Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 4 months...
- Ability to understand study procedures and to comply with them for the entire length of the study.
- Patients diagnosed with Low-Risk True Skin Only or a Single Bone lesion that does not require treatment and will only be observed will not be eligible, with the exception of CNS-risk lesions/special site disease or...
- CNS-risk/special site includes: Sphenoid, Mastoid, Orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease, odontoid peg, vertebral lesion with...
- Functionally critical: A single lesion not described above which may cause "functionally critical anatomic abnormality" wherein attempts at local therapy would cause unacceptable morbidity. This can be at the discretion...
- Patients whose genetic testing reveals a class 3 MAP2K1 mutation:
- I103\_K104del
- E102\_I103del
- L98\_K104delinsQ
- L98\_I103del
- I99\_K104del
- Patients who present with jaundice at diagnosis.
- Patients who are pregnant or breastfeeding are not eligible. Women of childbearing potential must receive a negative pregnancy test within 14 days of starting treatment or the patient will not be eligible.
- Patients who are allergic to trametinib
- Current drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements.
- Inability or unwillingness of patient or parent/legally authorized representative to give written informed consent.
The study team makes the final eligibility decision.
Where it's taking place
- Fort Worth, Texas, 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, 1 year to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Fort Worth, Texas, 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.