[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"langerhans-cell-histiocytosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:langerhans-cell-histiocytosis":208},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,48,76,99,130,158,182],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100509575","histiocytic-disorder-follow-up-study-100509575",false,"NCT05915208","Histiocytic Disorder Follow-up Study","Inclusion Criteria:\n\n* diagnosis of histiocytic disorder at any age\n\n  1. Langerhans cell histiocytosis,\n  2. Erdheim-Chester disease,\n  3. Rosai-Dorfman disease,\n  4. Xanthogranuloma,\n  5. Mixed histiocytosis\n  6. Malignant histiocytosis (Histiocytic sarcoma, langerhans cell sarcoma, interdigitating cell sarcoma)\n  7. Hemophagocytic lymphohistiocytosis\n\nExclusion Criteria:\n\n* None","ALL","0 Years","89 Years",{"count":19,"type":20},6000,"ESTIMATED","OBSERVATIONAL","The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders",[24,25,26,27,28,29],"Histiocytosis","Langerhans Cell Histiocytosis","Erdheim-Chester Disease","Rosai Dorfman Disease","Xanthogranuloma","Malignant Histiocytoses",[31,32,33,34],"survivorship","long-term","targeted therapy","second cancer","RECRUITING","2026-06-10",{"date":38,"type":39},"2026-06-12","ACTUAL",{"date":41,"type":39},"2022-09-01",{"date":43,"type":20},"2028-12",{"name":45,"class":46},"University of Alabama at Birmingham","OTHER",1,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":15,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":59,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":47},"100560868","phase-2-targeted-approach-to-langerhans-cell-histiocytosis-lch-using-mek-inhibitor-trametinib-100560868","NCT06582745","Targeted Approach to Langerhans Cell Histiocytosis (LCH) Using MEK Inhibitor, Trametinib","Inclusion Criteria:\n\n* Diagnosis\u002Fdisease status:\n\n  * Patients with newly diagnosed Langerhans cell histiocytosis (LCH) OR\n  * Patients with relapsed or refractory disease OR\n  * Patients with newly diagnosed or relapsed\u002Frefractory disease who are receiving the liquid formula of trametinib OR\n  * 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 receiving trametinib as treatment.\n* Diagnosis confirmed with biopsy prior to start of treatment\n* Patient must have adequate cardiac function evident through Echocardiogram (ECHO) and Electrocardiogram (EKG) within 30 days of starting treatment.\n\n  * Shortening fraction of ≥ 27% by echocardiogram or\n  * Ejection fraction of ≥ 50% by gated radionuclide study\n  * QTC \\\u003C 480 msec\n* 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.\n* Adequate organ and marrow function as defined below:\n\n  * Absolute Neutrophil count ≥ 1,500\u002FμL\n  * Platelets ≥ 100x103\u002FμL\n  * Total bilirubin ≤ 1.5X ULN for age\n  * AST\u002FALT ≤ 2.5 X ULN for age\n  * Serum creatinine based on age\u002Fgender\n  * Hemoglobin ≥ 8 g\u002FdL\n\n    * Patients with bone marrow disease must have hemoglobin ≥ 8 g\u002FdL with transfusion support allowed\n* 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 after the last dose. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.\n* Ability to understand study procedures and to comply with them for the entire length of the study.\n\nExclusion Criteria:\n\n* 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\u002Fspecial site disease or functionally critical lesions:\n\n  * CNS-risk\u002Fspecial site includes: Sphenoid, Mastoid, Orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease, odontoid peg, vertebral lesion with intraspinal soft tissue extension\n  * 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 of the Principal Investigator.\n* Patients whose genetic testing reveals a class 3 MAP2K1 mutation:\n\n  * I103\\_K104del\n  * E102\\_I103del\n  * L98\\_K104delinsQ\n  * L98\\_I103del\n  * I99\\_K104del\n* Patients who present with jaundice at diagnosis.\n* 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.\n\n  * Patients who are allergic to trametinib\n  * Current drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements.\n* Inability or unwillingness of patient or parent\u002Flegally authorized representative to give written informed consent.","1 Year","30 Years",{"count":57,"type":20},75,"INTERVENTIONAL",[60],"PHASE2","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.",[25],[64,65,66],"LCH","MEK inhibitor","Targeted therapy","2026-04-01",{"date":69,"type":39},"2026-04-07",{"date":71,"type":39},"2024-06-24",{"date":73,"type":20},"2039-12",{"name":75,"class":46},"Cook Children's Health Care System",{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":15,"minAge":83,"maxAge":84,"enrollmentInfo":85,"targetDuration":4,"studyType":58,"phases":87,"briefSummary":88,"conditions":89,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":47},"100607333","phase-2-efficacy-and-safety-of-low-dose-cytarabine-combined-with-thalidomide-in-adult-patients-with-untreated-lch-100607333","NCT07187193","Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH","Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in the Treatment of Adult Patients With Untreated Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study.","Inclusion Criteria:\n\n* Clearly diagnose and treat adult patients with multisystem or single-system multifocal LCH;\n* Aged between 18 and 70 years;\n* Multisystem involvement or single-system multifocal disease;\n* No prior systemic treatment (patients who have only received local radiotherapy or surgery may be enrolled);\n* ECOG performance status score ≤ 2;\n* Judged by clinicians as suitable for treatment with this protocol;\n* Patients or their families able to understand the study protocol and willing to participate in the study, providing written informed consent.\n\nExclusion Criteria:\n\n* Patients with LCH involving the central nervous system;\n* Single-system single-lesion LCH;\n* Subjects who have undergone major surgery within 4 weeks prior to the first dose in the study;\n* Subjects who have received radiotherapy within 4 weeks prior to the first dose in the study; subjects with a history of myocardial infarction within the past year;\n* Patients with New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or a history of NYHA class 3 or 4 congestive heart failure;\n* Pregnant or lactating women;\n* Abnormal liver and kidney function: creatinine level ≥176.8μmol\u002Fl (2mg\u002Fdl), transaminase and bilirubin levels more than 2 times the upper limit of normal (for LCH patients with liver involvement, bilirubin levels more than 10 times the upper limit of normal);\n* Abnormal blood counts: absolute neutrophil count less than 1×10\\^9\u002FL, platelet count less than 50×10\\^9\u002FL;\n* Patients or their families unable to understand the conditions and objectives of this study;\n* Any other circumstances in which the investigator deems the patient unsuitable to participate in this trial.","18 Years","70 Years",{"count":86,"type":20},50,[60],"Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.",[25],"2025-09-16",{"date":92,"type":39},"2025-09-22",{"date":94,"type":39},"2025-04-12",{"date":96,"type":20},"2027-04-13",{"name":98,"class":46},"Cancer Institute and Hospital, Chinese Academy of Medical Sciences",{"id":100,"slug":101,"hasResults":11,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":105,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":107,"targetDuration":4,"studyType":58,"phases":109,"briefSummary":110,"conditions":111,"keywords":117,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":129},"100368581","phase-2-cobimetinib-in-refractory-langerhans-cell-histiocytosis-lch-and-other-histiocytic-disorders-100368581","NCT04079179","Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders","A Phase 2 Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders.","NACHO-COBI","INCLUSION CRITERIA:\n\nAge at study entry\n\n* For Group 1: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment\n* For Group 2: Participant may be at least 6 months of age at the time of enrollment\n* For Group 3: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment\n* For Group 4: Participant must be 21 years of age or older at the time of enrollment\n* 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 or gastric tube.\n* Biopsy proven LCH -AND\n* Failure of at least front-line therapy for LCH with evaluable disease. -OR\n* Diagnosis of LCH-associated neurodegenerative disease with radiologic or clinical progression within the past 3 months. -OR\n* Biopsy proven JXG, ECD, RDD, histiocytic sarcoma, or other histiocytic lesion (newly diagnosed or relapsed\u002Frefractory disease) with evaluable active disease.\n\nPerformance Level:\n\n-Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50% for patients ≤ 16 years of age.\n\nAdequate Hematologic Function Defined as:\n\n* ANC ≥ 0.75 x 10\\^9\u002FL (unsupported\u002Fwithout growth factor stimulant)\n* Platelet count ≥ 75 x 10\\^9\u002FL (unsupported\u002Fwithout transfusion within the past 7 days).\n* Patients with marrow disease must have platelet count of \\>\u002F= 75 x 10\\^9\u002FL (transfusion support allowed) and must not be refractory to platelet transfusions.\n* Hemoglobin ≥ 8 g\u002FdL (unsupported\u002Fwithout transfusion within the past 7 days)\n* Patients with marrow disease must have hemoglobin ≥ 8 g\u002FdL (transfusion support allowed).\n\nAdequate Renal Function Defined as:\n\n\\- Calculated creatinine clearance (or radioisotope GFR) ≥ 70 mL\u002Fmin\u002F1.73m\\^2 or serum creatinine based on age\u002Fgender as follows:\n\nMaximum Serum Creatinine (mg\u002FdL) Age 2 to \\\u003C 6 years: Male 0.8 mg\u002FdL, Female 0.8; 6 to \\\u003C 10 years: Male 1 mg\u002FdL,Female 1; 10 to \\\u003C 13 years: Male 1.2 mg\u002FdL; Female 1.2; 13 to \\\u003C 16 years: Male 1.5 mg\u002FdL ; Female 1.4; ≥ 16 years: Male 1.7 mg\u002FdL; Female 1.4;\n\nAdequate Liver Function Defined as:\n\n* Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age\n* AST and ALT ≤ 3x ULN (≤ 5 x ULN for participants with liver involvement)\n* Serum albumin ≥ 2 g\u002FdL.\n\nFor patients with liver disease caused by histiocytic disorder:\n\n• Patients may be enrolled with abnormal bilirubin, AST, ALT and albumin with documentation of histiocytic liver disease.\n\nAdequate Cardiac Function Defined as:\n\n\\- Fractional shortening (FS) of ≥ 30% or ejection fraction of ≥ 50% by echocardiogram at baseline, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to enrollment. Depending on institutional standard, either FS or LVEF is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above\n\nPregnancy\u002FBirth Control\n\n* 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.\n* 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 partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) must agree to use two forms of effective methods of contraception (one of which must be a barrier method) during the treatment period and for at least 3 months after the last dose of the study drug to avoid pregnancy and\u002For potential adverse effects on a developing embryo. Agreement to true abstinence (not periodic abstinence or withdrawal method) is an acceptable method of birth control.\n\nEXCLUSION CRITERIA:\n\n\\- Prior and Concomitant Use of Drugs with CYP3A4 inducing\u002Finhibiting 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.\n\n* 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 enrollment, 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). Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the laboratory eligibility criteria are met, the patient is considered to have recovered adequately. See below for specific consideration of prednisone and corticosteroids prior to enrollment.\n\n  * Radiation therapy within the 14 days prior to enrollment.\n  * Any prior treatment with Cobimetinib.\n  * 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.\n  * Treatment with hormonal therapy (except hormone replacement therapy or oral contraceptives), immunotherapy, biologic therapy, investigational therapy, or herbal cancer therapy within 28 days or \\\u003C 5 half-lives, whichever is longer, prior to study enrollment.\n  * 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 receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial.\n  * For patients with brain tumors (intracranial masses), use of anticoagulants within 7 days prior to enrollment.\n  * Corticosteroid therapy less than or equal to 0.5 mg\u002Fkg\u002Fday 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 prior to enrollment and discontinue once study treatment has started.\n  * Patient has received treatment with investigational therapy within 4 weeks prior to initiation of study drug.\n  * Patients taking anticoagulants or have a pre-existing bleeding disorder unrelated to histiocytic disease.\n* Exclusions for other illness\n\n  * Other active malignancy or history of secondary malignancy.\n  * Refractory nausea and vomiting, malabsorption, external biliary shunt\n  * 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.\n  * 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 minor surgery is permitted within fourteen (14) days prior to study enrollment (provided that the wound has healed).\n  * History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease.\n  * History of pneumonitis.\n  * 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 (RVO), retinal detachment, retinal pathology on ophthalmologic exam, retinopathy of prematurity, central serous chorioretinopathy (CSSCR), neovascular retinopathy, intraocular pressure \\> 21 mmHg, and predisposing factors to RVO (e.g., uncontrolled hypertension, diabetes, or hyperlipidemia, coagulopathy) will be excluded. Patients with longstanding and stable ophthalmologic findings secondary to existing conditions are eligible with appropriate written documentation and approval from Study Chair.\n  * History of solid organ transplantation: Patients who have received a prior solid organ transplantation are not eligible.\n  * 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.\n* History of clinically significant cardiac dysfunction, including the following:\n\n  * Clinically significant cardiac arrhythmias including brady-arrhythmias and\u002For patients who require anti-arrhythmic therapy (with the exception of beta blockers or digoxin). Patients with controlled atrial fibrillation are not excluded.\n  * Unstable arrhythmia\n  * Unstable angina, or new-onset angina within 3 months prior to initiation of study treatment\n  * Symptomatic congestive heart failure, defined as New York Heart Association Class II or higher\n  * Myocardial infarction within 3 months prior to initiation of study treatment\n* Known chronic human immunodeficiency virus (HIV).\n* History of Grade ≥ 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of enrollment.\n* 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.",{"count":108,"type":20},90,[60],"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. Four different groups of patients will be enrolled.",[112,113,26,27,114,115,116],"Langerhan's Cell Histiocytosis","Juvenile Xanthogranuloma","Neuro-Degenerative Disease","Histiocytic Sarcoma","Histiocytic Disorders, Malignant",[118,119],"Cobimetinib","Langerhans Cell Histiocytosis (LCH)","2025-09-12",{"date":122,"type":39},"2025-09-18",{"date":124,"type":39},"2021-04-19",{"date":126,"type":20},"2029-12",{"name":128,"class":46},"Carl Allen",12,{"id":131,"slug":132,"hasResults":11,"nctId":133,"briefTitle":134,"officialTitle":135,"acronym":4,"eligibilityCriteria":136,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":137,"enrollmentInfo":138,"targetDuration":4,"studyType":58,"phases":140,"briefSummary":142,"conditions":143,"keywords":144,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":157},"100260534","phase-3-vinblastineprednisone-versus-single-therapy-with-cytarabine-for-langerhans-cell-histiocytosis-lch-100260534","NCT02670707","Vinblastine\u002FPrednisone Versus Single Therapy With Cytarabine for Langerhans Cell Histiocytosis (LCH)","Randomization of Cytarabine Monotherapy Versus Standard-of-Care Vinblastine\u002FPrednisone for Frontline Treatment of Langerhans Cell Histiocytosis (TXCH LCH0115)","Inclusion Criteria:\n\n1. Patient must have biopsy-confirmed diagnosis of Langerhans cell histiocytosis.\n2. Patient must be between 0-21 years of age.\n3. Patient must have a Karnofsky performance score ≥ 50% or Lansky performance score ≥ 50%.\n\nExclusion Criteria:\n\n1. Patient may not have received any prior systemic cytotoxic or other chemotherapies for LCH or any other malignant disorder prior to the initiation of protocol therapy on TXCH LCH0115 with the exception of:\n\n   Steroid pretreatment: Systemic glucocorticosteroids (prednisone, methylprednisone, dexamethasone, etc.) for less than or equal to 120 hours (5 days) in the 7 days prior to initiating protocol therapy or for less than or equal to 336 hours (14 days) in the 28 days before the initiation of protocol therapy does not affect eligibility. The dose of steroid previously given does not affect eligibility. Patients who have only received surgical or radiation therapy, intralesional injection of steroids, inhalational steroids, systemic mineralocorticoids (hydrocortisone), or topical steroids may also be enrolled.\n2. Patient may not have disease limited to a single skin or bone site, with the following exceptions:\n\n   * Central Nervous System (CNS) risk lesions\u002Fspecial site disease: patients with single bone sites that are CNS-risk (sphenoid, mastoid, orbital, zygomatic, ethmoid, maxillary, or temporal bones, the cranial fossa, pituitary gland or neurodegenerative disease) or are \"special sites\" (odontoid peg, vertebral lesion with intraspinal soft tissue extension) require systemic therapy as standard of care and thus are eligible for the study.\n   * Functionally critical lesions: A single lesion not described above which may cause \"functionally critical anatomic abnormality\" wherein attempts at local therapy (such as surgical curettage or radiation) would cause unacceptable morbidity. These patients may be enrolled with written approval of the Coordinating Center PI or Vice-Chair and documentation of the rationale justifying systemic therapy.\n   * Asynchronous multisite LCH presentation: A patient may also have any single site of disease involvement at the time of enrollment if they previously had at least one other site of LCH disease in the past (which may have been treated with local therapy\u002Fsurgery as described), as long as no systemic therapy was previously given per protocol guidelines.\n3. Patient may not have severe renal disease (creatinine greater than 3 times normal for age OR creatinine clearance \\\u003C 50 ml\u002Fm2\u002F1.73m\\^2).\n4. Patient may not have severe hepatic disease (direct bilirubin greater than 3 mg\u002Fdl OR aspartate aminotransferase (AST) greater than 500 IU\u002FL), unless hepatic injury is due to LCH.\n5. Female patients may not be pregnant or breastfeeding.\n6. Patients of reproductive potential not willing to use an adequate method of birth control for the duration of the study.\n7. Patients who are HIV positive may not be enrolled.\n\nNOTE: Patients excluded for laboratory abnormalities or performance score only may be enrolled on the study with written approval from the Coordinating Center PI or Vice-Chair.","21 Years",{"count":139,"type":20},124,[141],"PHASE3","Langerhans Cell Histiocytosis (LCH) is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body. Langerhans cell histiocytosis (LCH) is a cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions.\n\nFor most patients with LCH, standard-of-care vinblastine\u002Fprednisone are used as front-line therapy while cytarabine therapy has been used as therapy for patients who develop recurrence. No alternate treatment strategy has been developed for frontline therapy in LCH.\n\nThe purpose of this research study is to compare previously used vinblastine\u002Fprednisone to single therapy with cytarabine for LCH. We will evaluate the utility of an imaging study called a positron emission tomography (PET) scan to more accurately assess areas of LCH involvement not otherwise seen in other imaging studies as well as response to therapy. We also want to identify if genetic and other biomarkers (special proteins in patient's blood and in patient's cancer) relate to the response of patients LCH to study treatment.",[25],[145,146,147,119],"vinblastine","prednisone","cytarabine","2025-09-05",{"date":150,"type":39},"2025-09-11",{"date":152,"type":39},"2016-03-07",{"date":154,"type":20},"2029-01",{"name":156,"class":46},"Baylor College of Medicine",11,{"id":159,"slug":160,"hasResults":11,"nctId":161,"briefTitle":162,"officialTitle":163,"acronym":4,"eligibilityCriteria":164,"healthyVolunteers":11,"sex":15,"minAge":165,"maxAge":166,"enrollmentInfo":167,"targetDuration":4,"studyType":58,"phases":169,"briefSummary":170,"conditions":171,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":172,"lastUpdatePostDateStruct":173,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":157},"100515906","phase-2-to-evaluate-the-efficacy-safety-and-pk-characteristics-of-fcn-159-in-pediatric-patients-with-refractoryrecurrent-lch-100515906","NCT05997602","To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory\u002FRecurrent LCH","A Multi-center, Open-label, Single Arm Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of FCN-159 in Pediatric Patients With Refractory\u002FRecurrent Langerhans Cell Histiocytosis","Inclusion Criteria:\n\n1. Age 2-16 (inclusive)\n2. Patients with histologically confirmed Langerhans cell histiocytosis (LCH) diagnosed by the central laboratory.\n3. If sufficient tumor tissue samples and peripheral blood samples are available, central laboratory biomarker testing is required as follows: including but not limited to ERBB3, BRAF, ARAF, HRAS, KRAS, NRAS, MEK (MAP2K1 and MAP2K2), and other MEK upstream genes.If inability to get tissue, the gene testing results from a local laboratory also can be accepted.\n4. Patients who have received at least prior first-line systemic treatment, defined as treatment including vinblastine (VBL) and glucocorticoids for at least 2 weeks. VBL can be substituted with vincristine (VCR) or vindesine (VDS). Alternatively, patients may be unable to tolerate chemotherapy due to severe chemotherapy toxicity. Inability to tolerate chemotherapy is defined as one of the following: Severe liver impairment (liver enzyme elevation ≥ 5 × upper limit of normal (ULN) and bilirubin elevation ≥ 1.5 × ULN), severe neurotoxicity related to vinca alkaloids, chemotherapy-related intracranial hypertension, or grade 4 bone marrow depression with severe infection (sepsis, severe pneumonia, etc.) after chemotherapy.\n5. Refractory\u002Frelapsed LCH is defined as the presence of one of the following:\n\n   1. Failure of prior treatment, i.e., no regression in risk organs after at least 2 weeks of systemic treatment, or overall evaluation of AD-progression or AD-mix;\n   2. Initial response of the disease to first or second-line systemic treatment is NAD or AD-better or AD-stable, followed by disease reactivation after maintenance therapy for more than 3 months. Second-line treatment includes cytarabine and\u002For cladribine.\n   3. Persistent mutated gene positive in plasma free DNA testing during prior treatment (confirmed by 2 consecutive tests) or retest positive after treatment discontinuation;\n   4. Lack of regression in the affected central nervous system (including the pituitary gland) after treatment;\n   5. Presence of bone marrow involvement and\u002For hemophagocytic lymphohistiocytosis (HLH);\n6. Presence of evaluable lesions based on PET response criteria (PRC).\n7. Patients who have to have recovered from all acute toxic effects of prior anti-tumor therapy, and all relevant toxicities must be ≤ grade 1 (except for alopecia and ototoxicity).\n8. Expected survival at least ≥ 3 months;\n9. Lansky (≤ 15 years old) and Karnofsky (≥ 16 years old) performance status scores should be ≥ 50%, as shown in Appendix 4.\n10. Patients or their legal guardians must be able to understand and willingly sign a written informed consent form.\n11. For women of childbearing potential, a serum human chorionic gonadotropin (HCG) pregnancy test must be negative within 7 days before starting treatment.\n12. For female patients of childbearing potential: Patients should agree to use effective contraception methods during the treatment period and for at least 90 days after the last dose of study treatment, using dual barrier contraception methods such as condoms, oral or injectable contraceptives, intra-uterine contraceptive devices, etc. Male patients should agree to refrain from donating sperm for at least 90 days after the last dose of study treatment.\n13. Adequate bone marrow function: Absolute neutrophil count ≥ 1.0×10\\^9\u002FL, hemoglobin ≥ 90g\u002FL, and platelets ≥ 75×10\\^9\u002FL without the use of blood transfusions, blood products, or granulocyte colony-stimulating factors. Patients with hematocytopenia below these thresholds due to the underlying disease may be considered for inclusion based on the investigator's comprehensive judgment.\n14. Adequate hepatic and renal function: Serum total bilirubin ≤ 1.5 × the upper limit of normal (ULN), or ≤ 5× ULN for patients with Gilbert's syndrome or liver involvement; aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (AKP) ≤ 2.5 × ULN, or ≤ 10 × ULN for patients with liver involvement; albumin ≥ 3g\u002FdL; and creatinine clearance or isotopic glomerular filtration rate (GFR) ≥ 50ml\u002Fmin\u002F1.73㎡or serum creatinine based on age; hepatic and renal impairment caused by the primary disease may be considered for inclusion based on the investigator's comprehensive judgment.\n15. Coagulation: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 ULN.\n\nExclusion Criteria:\n\n1. Patients who have received any of the following prior treatments:\n\n   1. Chemotherapy, targeted therapy, immunotherapy, biologic therapy, or herbal anti-tumor therapy for LCH within 4 weeks or \\\u003C 5 half-lives (whichever is shorter)before the start of the study drug .\n   2. Strong CYP3A4, CYP2C8, and CYP2C9 inhibitors or inducers within 14 days before the start of the study drug, except for topical skin application.\n   3. Gowth factors that promote platelet or white blood cell count or function within 7 days before the start of the study drug.\n   4. Radiotherapy or major surgical treatment (including craniotomy, thoracotomy, laparotomy, open bone or joint surgery, etc.) within 4 weeks before the start of the study drug.\n   5. Participated in other interventional clinical trials within 4 weeks before the start of the study drug.\n   6. MEK 1\u002F2 inhibitors (those who have received this treatment for a short period of ≤ 2 weeks may be included).\n   7. Anticoagulants within 7 days before the start of the study drug for patients with brain tumors (intracranial masses).\n   8. Prednisone treatment \\\u003C 0.5mg\u002Fkg\u002Fday (or equivalent dose of other corticosteroids) is allowed within one month before enrollment, but must be discontinued 14 days before the start of the study drug. Patients with brain lesions receiving corticosteroid therapy for brain edema must maintain a stable dose for 14 days before enrollment. Hormone replacement therapy is allowed for patients with hypopituitarism due to primary disease involvement of the pituitary.\n2. Patients with a history of other malignant tumors or concurrent other malignant tumors (excluding cured non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ).\n3. Uncontrolled hypertension (with medication treatment): Blood pressure (BP) greater than or equal to the 95th percentile for age, height, and sex, as described in Appendix 6.\n4. Patients with dysphagia, active gastrointestinal disease, malabsorption syndrome, or other conditions that may affect the absorption of the study drug.\n5. Prior or current history of retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other clinically significant abnormal ophthalmologic examination results.\n6. Interstitial pneumonia, including clinically significant radiation pneumonitis. Except for interstitial pneumonia caused by pulmonary involvement of the primary disease.\n7. Patients will be excluded if their cardiac function or comorbidities meet any of the following criteria:\n\n   1. During the screening period, 12-lead electrocardiogram (ECG) measurements will be taken three times at the study center with a mean value calculated using the QTcF formula provided by the instrument; patients with a mean value of QTcF \\> 470 milliseconds or with risk factors for QTcF prolongation, such as uncorrected hypokalemia, congenital long QT syndrome, or receiving drugs known to prolong QTcF interval (mainly class Ia, Ic, and III antiarrhythmic drugs) will be excluded from the study. Drugs with the potential to prolong the QTcF interval are listed in Appendix 7.\n   2. New York Heart Association (NYHA) Class 2 and above congestive heart failure as shown in Appendix 5.\n   3. Clinically significant arrhythmias, including but not limited to complete left bundle branch block, and second-degree atrioventricular block.\n   4. Known presence of clinically significant coronary heart disease, cardiomyopathy, or severe valvular disease.\n   5. Echocardiography examination indicating left ventricular ejection fraction (LVEF) \\\u003C 50%.\n8. Patients with active bacterial, fungal, or viral infections, including active hepatitis B (defined as positive hepatitis B surface antigen and hepatitis B virus DNA \\> 1000IU\u002Fml or meeting the diagnostic criteria for active hepatitis B infection at the study center) or hepatitis C (positive hepatitis C virus RNA), or human immunodeficiency virus (HIV positive) infection.\n9. Patients with known allergies to the study drug, other MEK1\u002F2 inhibitors, or their excipients.\n10. Patients with known tumor tissue genetic testing that indicates the presence of MAP2K1 exon 3 deletions (del) or deletion-insertion type (delins\u002Findels) mutations.\n11. The investigator considers clinically significant cases that will impede participation in the study or prevent compliance with safety requirements.","2 Years","16 Years",{"count":168,"type":20},56,[60],"This is a rare disease, single-arm, open-label,multi-center, non-randomized Phase 2 clinical study to evaluate the efficacy, safety, and pharmacokinetic characteristics of FCN-159 monotherapy in pediatric patients with refractory\u002Frecurrent Langerhans cell histiocytosis (LCH).",[25,64],"2025-01-22",{"date":174,"type":39},"2025-01-24",{"date":176,"type":39},"2023-09-28",{"date":178,"type":20},"2027-05-14",{"name":180,"class":181},"Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.","INDUSTRY",{"id":183,"slug":184,"hasResults":11,"nctId":185,"briefTitle":186,"officialTitle":187,"acronym":4,"eligibilityCriteria":188,"healthyVolunteers":11,"sex":15,"minAge":189,"maxAge":83,"enrollmentInfo":190,"targetDuration":4,"studyType":58,"phases":192,"briefSummary":193,"conditions":194,"keywords":195,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":199,"lastUpdatePostDateStruct":200,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":47},"100421866","phase-3-a-prospective-study-for-the-treatment-of-children-with-newly-diagnosed-lch-using-a-cytarabine-contained-protocol-100421866","NCT04773366","A Prospective Study for the Treatment of Children With Newly Diagnosed LCH Using a Cytarabine Contained Protocol","A Prospective Institutional Study for the Treatment of Children With Newly Diagnosed Langerhans Cell Histiocytosis Using a Cytarabine Contained Protocol","Inclusion Criteria:\n\n1. Age under 18 years\n2. Newly diagnosed LCH：Morphologic identification of the characteristic LCH cells, positive staining of the lesional cells with CD1α and\u002For Langerin\n3. No congenital immunodeficiency, HIV infection, or prior organ transplant\n4. No previous chemotherapy\u002Ftarget therapy\u002Fradiation, if any steroid applied, total prior steroids dosage \\\u003C prednisone 280 mg\u002Fm2\n\nExclusion Criteria:\n\n* Patients have overwhelming infection, and a life expectancy of \\\u003C 2 weeks","1 Day",{"count":191,"type":20},200,[141],"From January 2010 to December 2014, 150 children with MS-LCH were treated in our hospital following a LCH II (Arm B) based protocol. Treatment was based on a modification of the LCH-II (Arm B) based protocol. However, the continuation treatment was extended to 56 weeks and etoposide was omitted from the continuation treatment.\n\nFor the 59 patients with RO involvement (RO+) (the lungs are not considered a RO in the current study), the rapid response rate (week 6) was 61.0% and the 3-year overall survival (OS) 73.4±5.9%. Rapid responders had a better 3-year survival rate than poor responders (90.9±5.0% vs. 45.7±11.0%, P\\\u003C0.001). The 3-year OS in the current study is 10\\~20% lower than the rates reported by Gadner et al. and Morimoto et al.. We have not yet adopted effective salvage therapies for RO+ patients with recurrent disease. During the time of this study, cladribine was unavailable. Second-line therapy for non-responders or patients with disease reactivation was individualized treatment based on the physician's experience. An effective salvage therapy is essential for this high-risk group.\n\nFor 91without RO involvement (RO-), 78 patients (85.7%) were rapid responders at week 6. The 3-year cumulative reactivation rate was 10.7% for RO- patients. No death occurred in this subgroup, with a 3-year OS of 100% in RO- patients. Compared to the LCH II and LCH III trials, the current study had a more intensive initial treatment regimen for RO- patients. However, the addition of etoposide to prednisone and vincristine in the initial therapy did not increase the 6-week response rate for RO- patients (85.7% in this study compared to 83% in the LCH II study and 86% in the LCH III study). Surprisingly, with a relatively intense initial treatment, a relatively low 3-year cumulative reactivation rate was observed in RO- patients in the current study. This result suggests that the initial treatment intensity and duration of continuation therapy both impact disease reactivation. The intensity of induction can affect the degree of disease resolution. Insufficient treatment intensity might lead to late relapse. Similarity to that observed has been in other childhood hematological malignancies. This finding deserves to be tested in prospective clinical trials with long-term follow-up. Cytarabine has been applied for patients with LCH but has never been evaluated in our hospital prospectively. In this study, we administer a cytarabine contained protocol to patients with multisystem involvement with or without risk organs involvement. The treatment results will be compared with our historical studies.",[25],[196,197,147,198],"Langerhans cell histiocytosis","pediatric patients","treatment outcome","2022-07-23",{"date":201,"type":39},"2022-07-26",{"date":203,"type":39},"2018-07-01",{"date":205,"type":20},"2026-06-30",{"name":207,"class":46},"Shanghai Children's Medical Center","Langerhans-Cell Histiocytosis"]