Learn about Research & Clinical Trials
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||N/A and Over|
- - For Group 1: Participant must be less than 30 years of age at the time of enrollment.
- - For Group 2: Participant may be any age at the time of enrollment.
- - For Group 3: Participant must be less than 30 years of age at the time of enrollment.
- - For Group 4: Participant must be 30 years of age or older at the time of enrollment.
- - Participant must be able to take an enteral dose and formulation of medication.
- - Biopsy proven LCH -AND.
- - Failure of at least front-line therapy for high-risk LCH with evaluable disease.
- - Failure of at least second-line therapy for low-risk LCH with evaluable disease.
- - Diagnosis of LCH-associated neurodegenerative disease with radiologic or clinical progression within the past 3 months.
- - Biopsy proven JXG, ECD, RDD, histiocytic sarcoma, or other histiocytic lesion (newly diagnosed or relapsed/refractory disease) with evaluable active disease.
- - 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).
- - Calculated creatinine clearance (or radioisotope GFR) ≥ 70 mL/min/1.73m^2 or serum creatinine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age 2 to < 6 years: Male 0.8 mg/d, Female 0.8; 6 to < 10 years: Male 1 mg/dL,Female 1; 10 to < 13 years: Male 1.2 mg/dL; Female 1.2; 13 to < 16 years: Male 1.5 mg/dL ; Female 1.4; ≥ 16 years: Male 1.7 mg/dL; Female 1.4; Adequate Liver Function Defined as: - Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age.
- - AST and ALT ≤ 2.5x ULN for age.
- - Serum albumin ≤ 2 g/dL.
- - Fractional shortening (FS) of ≥ 30% or ejection fraction of ≥ 50% by echocardiogram at baseline, as determined by echocardiography or multigated acquisition scan (MUGA) within 21 days prior to study database registration.
- - 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.
- - 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 following: erythromycin, clarithromycin, ketoconazole, azithromycin, itraconazole, grapefruit juice or St. John's wort.
- - 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 database registration, with resolution of all associated toxicity to ≤ Grade 1 prior to study enrollment (exception for alopecia and ototoxicity which do not need to be resolved ≤ Grade 1).
- - Radiation therapy within the last 28 days.
- - 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 database registration.
- - 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, whichever is longer, prior to database registration.
- - Treatment with high-dose chemotherapy and stem-cell rescue (autologous stem cell transplant) or allogeneic stem cell transplant within 90 days prior to study database registration.
- - For patients with brain tumors (intracranial masses), use of anticoagulants within 7 days prior to study database registration.
- - Corticosteroid therapy <0.5 mg/kg/day averaged during the month prior to study database registration is permissible but must be discontinued fourteen (14) days prior to prior to study database registration.
- - Patient has received treatment with investigational therapy within 4 weeks prior to initiation of study drug.
- - 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 study database registration that has not completely resolved.
- - Major surgical procedure or significant traumatic injury within 28 days prior to study database registration, or anticipation of need for major surgical procedure during the course of the study.
- - 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.
- - 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 contraindicates use of an investigational drug or places the patient at unacceptable risk from treatment complications.
- - 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).
- - 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 study entry.
- - Female patients who are pregnant or lactating.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Carl E Allen, MD, PhD|
|Principal Investigator Affiliation||Baylor College of Medicine|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Langerhan's Cell Histiocytosis, Juvenile Xanthogranuloma, Erdheim-Chester Disease, Rosai Dorfman Disease, Neuro-Degenerative Disease, Histiocytic Sarcoma, Histiocytic Disorders, Malignant|
Histiocytic disorders are diseases caused by misfunctioning or buildup of particular immune cells called histiocytes. Many histiocytic disorders (LCH, juvenile xanthogranuloma (JXG), Erdheim-Chester disease (ECD), and Rosai-Dorfman Disease (RDD)) arises from blood cells that receive incorrect growth signals. These incorrect signals are caused by changes in genes (mutations) that lead to tissue damage (lesions) which causes disease. Some patients with LCH can develop neurodegeneration (LCH-ND) which is damage to neurons that results in reduced brain function, from LCH cells that go to the brain and activate inflammation. LCH arises from blood cells that receive incorrect growth signals. These incorrect signals are caused by mutations (changes in genes). The LCH blood cells can create changes in the structure of almost any organ, and can cause damage to normal organ function. The purpose of this research study is to learn whether cobimetinib is safe and effective in subjects diagnosed with LCH, LCH-ND, RDD, JXG and ECD which may have a specfic mutation called BRAF-V600E. In healthy cells, certain proteins (called BRAF and MEK) are thought to help control normal cell growth. BRAF-V600E is a specific change in a gene that may cause cancer cells to grow and spread by sending constant signals to the MEK protein. Cobimetinib is designed to attach to and block the activity of MEK.
Experimental: Patients < 30 years with recurrent LCH (Grp1)
Children and young adults (<30 years) with recurrent active LCH lesions (may also have LCH-ND).
Experimental: Patients of any age with LCH-ND (Grp2)
Patients of any age with progressive LCH Neurodegenerative Disease (LCH-ND) without other sites of active LCH.
Experimental: Patients <30 years with other histiocytic disorders (Grp3)
Newly diagnosed or relapsed/refractory children and young adults (<30 years) with other histiocytic disorders including juvenile xanthogranuloma, Erdheim-Chester disease, histiocytic sarcoma and Rosai-Dorfman disease.
Experimental: Patients ≥ 30 years with LCH/histiocytic disorders (Grp4)
Adults (≥30 years) with LCH or other histiocytic disorder with recurrent active lesions (may also have LCH-ND).
Drug: - Cobimetinib
All groups will receive the same intervention. Cobimetinib will be given orally at a maximum dose of 60mg daily for 21 days on, then 7 days off, in a 28-day treatment cycle for a total of 12 cycles (approximately 12 months).
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.