Langerhans cell histiocytosis
Conditions
Interventions
Group 1:Ranadomide
Group 2:MEK Inhibitor
Group 3:Renadomide combined with MEK inhibitor
Sponsors
Department of Oncology, Shanghai Sixth People's Hospital
Eligibility
Sex/Gender
Male
Age
14 Years to 70 Years
Inclusion criteria
Inclusion criteria: 1. The patients volunteered to participate in the study and signed the informed consent; 2. Aged 14-70 years old, the body surface area of patients under 18 years old should be > 1.5m2; ECOG PS score: 0-1; the estimated survival time is more than 3 months; 3. Pathologically diagnosed as Langerhans histiocytosis of bone; 4. Patients who fail to receive at least one routine treatment (including cytotoxic drugs and/or hormone therapy) and whose disease progresses or is intolerable within six months before admission; Note: 1. Treatment failure refers to disease progression or intolerance during or within 3 months of the last treatment; (1) Blood routine examination criteria (14 days without blood transfusion): Hemoglobin (HB) > 90g/L; Neutrophil absolute value (ANC) > 1.5 *10^9/L; Platelet (PLT) > 80 *10^9/L; (2) Biochemical tests should meet the following criteria: Total bilirubin (TBIL) less than 1.5 times the upper limit of normal value (ULN); Alanine aminotransferase (ALT) and aspartate aminotransferase AST 60 ml/min; (3) Doppler echocardiography: left ventricular ejection fraction (LVEF) > normal low limit (50%); 5. Women of reproductive age should agree that contraceptive measures (such as intrauterine devices, contraceptives or condoms) must be used within 6 months of the study period and the end of the study; serum or urine pregnancy tests should be negative within 7 days prior to the study and must be non-lactating; and men should agree that contraceptive measures must be used within 6 months of the study period and the end of the study period.
Exclusion criteria
Exclusion criteria: Patients with any of the following items will not be eligible for this study: 1. Respiratory syndrome with pleural effusion/ascites (according to NCI CTC AE grade (> 2 grade dyspnea). [Grade 2 dyspnea refers to shortness of breath when a small amount of activity affects instrumental activities of daily life]; 2. Other malignant tumors occurred within 5 years or are currently concurrent with other malignant tumors, except cervical carcinoma in situ, skin cancer without melanoma and superficial bladder tumors [Ta (non-invasive tumors), Tis (carcinoma in situ) and T1 (invasive basement membrane of tumors)]; 3. Systemic antineoplastic therapy, including cytotoxic therapy, signal transduction inhibitors, immunotherapy (or mitomycin C, was planned within the first four weeks of treatment or during the current study). Over-extended-field radiotherapy (EF-RT) was performed in the first four weeks of grouping or limited-field radiotherapy was performed in the first two weeks of grouping; 4. Unmitigated toxic reactions higher than CTC AE (4.0) level 1 or above due to any previous treatment, excluding hair loss; 5. Those with multiple factors affecting oral medicines (such as inability to swallow, chronic diarrhea, intestinal obstruction, etc.); 6. Patients with brain metastasis with symptoms or less than 2 months of symptom control; 7. Patients with any serious and/or uncontrollable disease, including: (1) Patients with unsatisfactory blood pressure control (systolic blood pressure (> 150 mmHg), diastolic blood pressure (> 100 mmHg); (2) Patients with grade I or above myocardial ischemia or myocardial infarction, arrhythmia (including QTc CTC AE 2 infection); (4) Liver cirrhosis, decompensated liver disease, active hepatitis or chronic hepatitis require antiviral treatment; (5) Renal failure requires hemodialysis or peritoneal dialysis; (6) Have a history of immunodeficiency, including HIV positive or other acquired, congenital immunodeficiency disorders, or organ transplantation history; (7) Poor control of diabetes mellitus (FBG) > 10 mmol/L); (8) Urinary routine showed that urinary protein (++) and 24-hour urinary protein quantification (>1.0 g) was confirmed; (9) Patients with epilepsy and need treatment; 8. Major surgical treatment, incision biopsy or obvious traumatic injury were performed within 28 days before the grouping; 9. The imaging findings showed that the tumors had invaded important perivascular areas or that the tumors were highly likely to invade important blood vessels and cause fatal massive hemorrhage during the follow-up study period; 10. No matter the severity, there are any physical signs or history of bleeding in patients. Within the first four weeks of the group, patients with any bleeding or bleeding event (> CTCAE grade 3) have unhealed wounds, ulcers or fractures; 11. Arteriovenous thrombosis events occurred within 6 months, such as cerebrovascular accident (including temporary ischemic attack), deep vein thrombosis and pulmonary embolism; 12. Those who have a history of psychotropic drug abuse and are unable to give up or have mental disorders; 13. Participated in clinical trials of other antineoplastic drugs within four weeks; 14. According to the judgement of the researcher, there are concomitant diseases that seriously endanger the safety of patie
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| WFDC21P;PFS; | — |
Secondary
| Measure | Time frame |
|---|---|
| ORR=CR+PR;DCR=CR+PR+SD;OS;Safety; | — |
Countries
China
Contacts
Public ContactTANG LINA
Department of Oncology, Shanghai Sixth People's Hospital
Outcome results
None listed