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New treatment option for Langerhan's Cell Histiocytosis
Official title Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
ClinicalTrials.gov ID: NCT04079179
What this study is testing
What is Cobimetinib?
Cobimetinib is an investigational medicine, given as an once-daily, being studied as a potential treatment for langerhan's cell histiocytosis.
Also referred to as COTELLIC, RO5514041.
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
- This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells.
- 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
Adults
You may be able to join if
- Age at study entry
- For Group 1: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
- For Group 2: Participant may be at least 6 months of age at the time of enrollment
- For Group 3: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
- For Group 4: Participant must be 21 years of age or older at the time of enrollment
You likely can't join if
- \- Prior and Concomitant Use of Drugs with CYP3A4 inducing/inhibiting activity: Patient taking strong inducers or inhibitors of CYP3A4 within 14 days...
- Prior Therapy Restrictions Completion of previous chemotherapy, immunotherapy, radiotherapy, or targeted therapy for LCH (or other histiocytic...
- Radiation therapy within the 14 days prior to enrollment.
- Any prior treatment with Cobimetinib.
- Treatment with a long-acting hematopoietic growth factor within 14 days prior to initiation of study drug or a short-acting hematopoietic growth...
- Treatment with hormonal therapy (except hormone replacement therapy or oral contraceptives), immunotherapy, biologic therapy, investigational...
See the full eligibility criteria
Who can join
- Age at study entry
- For Group 1: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
- For Group 2: Participant may be at least 6 months of age at the time of enrollment
- For Group 3: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
- For Group 4: Participant must be 21 years of age or older at the time of enrollment
- Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet which may be taken by mouth or other enteral route such as nasogastric...
- Biopsy proven LCH -AND
- Failure of at least front-line therapy for LCH with evaluable disease. -OR
- Diagnosis of LCH-associated neurodegenerative disease with radiologic or clinical progression within the past 3 months. -OR
- Biopsy proven JXG, ECD, RDD, histiocytic sarcoma, or other histiocytic lesion (newly diagnosed or relapsed/refractory disease) with evaluable active disease. Performance Level: -Karnofsky ≥ 50% for patients \> 16 years...
- ANC ≥ 0.75 x 10\^9/L (unsupported/without growth factor stimulant)
- Platelet count ≥ 75 x 10\^9/L (unsupported/without transfusion within the past 7 days).
- Patients with marrow disease must have platelet count of \>/= 75 x 10\^9/L (transfusion support allowed) and must not be refractory to platelet transfusions.
- Hemoglobin ≥ 8 g/dL (unsupported/without transfusion within the past 7 days)
- Patients with marrow disease must have hemoglobin ≥ 8 g/dL (transfusion support allowed). Adequate Renal Function Defined as: \- Calculated creatinine clearance (or radioisotope GFR) ≥ 70 mL/min/1.73m\^2 or serum...
- Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
- AST and ALT ≤ 3x ULN (≤ 5 x ULN for participants with liver involvement)
- Serum albumin ≥ 2 g/dL. For patients with liver disease caused by histiocytic disorder:
- Patients may be enrolled with abnormal bilirubin, AST, ALT and albumin with documentation of histiocytic liver disease. Adequate Cardiac Function Defined as: \- Fractional shortening (FS) of ≥ 30% or ejection fraction...
- Female patients of childbearing potential require a negative urine or serum pregnancy test for eligibility and again at database registration, if more than 2 weeks has elapsed.
- Female patients of childbearing potential must agree to follow the contraceptive requirements using two forms of effective contraceptive methods for the duration of the study treatment. Male patients with sexual...
What rules you out
- \- Prior and Concomitant Use of Drugs with CYP3A4 inducing/inhibiting activity: Patient taking strong inducers or inhibitors of CYP3A4 within 14 days prior to study enrollment, including but not limited to the...
- Prior Therapy Restrictions Completion of previous chemotherapy, immunotherapy, radiotherapy, or targeted therapy for LCH (or other histiocytic disorder) at least 28 days (except where specified below) prior to study...
- Radiation therapy within the 14 days prior to enrollment.
- Any prior treatment with Cobimetinib.
- Treatment with a long-acting hematopoietic growth factor within 14 days prior to initiation of study drug or a short-acting hematopoietic growth factor within 7 days prior to enrollment.
- Treatment with hormonal therapy (except hormone replacement therapy or oral contraceptives), immunotherapy, biologic therapy, investigational therapy, or herbal cancer therapy within 28 days or \< 5 half-lives...
- Treatment with high-dose chemotherapy and stem-cell rescue (autologous stem cell transplant) or allogeneic stem cell transplant within 90 days prior to enrollment. Anti-GVHD agents post-transplant: Patients who are...
- For patients with brain tumors (intracranial masses), use of anticoagulants within 7 days prior to enrollment.
- Corticosteroid therapy less than or equal to 0.5 mg/kg/day averaged during the 28 days prior to study enrollment is permissible. Patients receiving corticosteroids must be on a stable or decreasing dose for 14 days...
- Patient has received treatment with investigational therapy within 4 weeks prior to initiation of study drug.
- Patients taking anticoagulants or have a pre-existing bleeding disorder unrelated to histiocytic disease.
- Exclusions for other illness
- Other active malignancy or history of secondary malignancy.
- Refractory nausea and vomiting, malabsorption, external biliary shunt
- Infection: Patients who have a known active infection (excluding documented fungal infection of the nail beds) within 28 days prior to enrollment that has not completely resolved.
- Major surgical procedure or significant traumatic injury within 28 days prior to enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or...
- History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease.
- History of pneumonitis.
- Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion are not eligible. Specifically, patients with a history of retinal vein occlusion...
- History of solid organ transplantation: Patients who have received a prior solid organ transplantation are not eligible.
- Any other disease, metabolic or psychological dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that in the opinion of the investigator...
- History of clinically significant cardiac dysfunction, including the following:
- Clinically significant cardiac arrhythmias including brady-arrhythmias and/or patients who require anti-arrhythmic therapy (with the exception of beta blockers or digoxin). Patients with controlled atrial fibrillation...
- Unstable arrhythmia
- Unstable angina, or new-onset angina within 3 months prior to initiation of study treatment
- Symptomatic congestive heart failure, defined as New York Heart Association Class II or higher
- Myocardial infarction within 3 months prior to initiation of study treatment
- Known chronic human immunodeficiency virus (HIV).
- History of Grade ≥ 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of enrollment.
- Female patients who are pregnant or lactating. Pregnant or lactating women will not be entered on this study because there is no available information regarding human fetal or teratogenic toxicities.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Little Rock, Arkansas, United States
- Orange, California, United States
- San Francisco, California, United States
- Washington D.C., District of Columbia, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Memphis, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Madison, Wisconsin, 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, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Little Rock, Arkansas, United States; Orange, California, United States; San Francisco, California, United States; Washington D.C., District of Columbia, United States; Baltimore, Maryland, United States and 6 more location(s). 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.