Neurofibromatosis type 1,plexiform neurofibromas
Conditions
Interventions
Experimental group:Oral FCN-159 tablets
Sponsors
Beijing Childrens Hospital,Capital Medical University
Eligibility
Sex/Gender
All
Age
2 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. >= 2 years old but= 3 centimeters in at least one dimension, which can be evaluated for effectiveness using MRI. 6. Karnofsky physical fitness score >= 70 points (applicable for those over 16 years old); 7. Or Lansky physical fitness score >= 70 points (applicable for = 1.0 × 10^9/L; Hemoglobin >= 90g/L; Platelets >= 75 × 10^9/L; Total serum bilirubin = 3g/dL; Creatinine = 50ml/min/1.73m2 (calculated using Cockcroft Gault formula); Urinary protein= 2+, the 24-hour urine protein quantification must be <= 1g. 8. Coagulation function: International standardized ratio (INR) and activated partial thromboplastin time (APTT) <= 1.5 × ULN. 9. The patient or their legal guardian is able to understand and voluntarily sign a written informed consent form.
Exclusion criteria
Exclusion criteria: 1. Malignant tumors related to NF1 that require chemotherapy, radiation therapy, or surgical treatment, such as high-grade glioblastoma or malignant peripheral schwannoma. 2. Has a history of other malignant tumors or is also suffering from other malignant tumors. 3. Inability to accept MRI examination and/or contraindications for MRI examination. 4. Difficulty in swallowing, active digestive system diseases, malabsorption syndrome, or other conditions that affect the absorption of investigational drugs. 5. Previous or current ophthalmic examinations with retinal vein occlusion (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other significant abnormalities. 6. Interstitial pneumonia, including clinically significant radiation pneumonia. 7. Cardiac function or comorbidities meet one of the following conditions: a. QTcF>470 milliseconds; Patients with QTcF prolongation risk factors, such as inability to correct hypokalemia and hereditary long QT syndrome; Or receive drugs that prolong the QTcF interval (mainly Ia, Ic, III class antiarrhythmic drugs). b. Congestive heart failure rated >= 3 by the New York Heart Association (NYHA) in the United States; Clinically significant arrhythmias, including but not limited to complete left bundle branch conduction abnormalities and second degree atrioventricular block; Known clinically significant complications include coronary heart disease, cardiomyopathy, and severe valve disease. e. The echocardiography showed that the left ventricular ejection fraction (LVEF) was less than 50%. f. Patients with bradycardia whose heart rate is less than 50 beats per minute. 8. There are immediate relatives in the family who experienced sudden cardiac death before the age of 50. Direct relatives are defined as upper and lower generations of relatives who have a hereditary blood relationship with each other, such as parents and children, grandparents and grandchildren, and grandparents and grandchildren. 9. Have any history of acute neurological disorders (i.e. intracranial or subarachnoid hemorrhage, cerebral infarction, intracranial trauma) within the 6 months prior to enrollment. 10. Associated with active bacterial, fungal or viral infection, including active hepatitis B (hepatitis B B virus surface antigen positive and hepatitis B virus DNA more than 1000 IU/ml or in line with the research center's diagnostic criteria for active hepatitis B infection), hepatitis C (hepatitis C virus RNA positive), human immunodeficiency virus infection (HIV positive). 11. Known to be allergic to the investigational drug, other MEK1/2 inhibitors, or their excipients. 12.The researcher believes that other situations are not suitable for participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Tumour size;Blood biochemistry;Coagulation;Urine tests;Routine blood tests; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pain intensity assessment;Cardiac ultrasound;Electrocardiogram indicators;Ophthalmic examination indicators; | — |
Countries
China
Contacts
Public ContactXin Ni
Beijing Childrens Hospital,Capital Medical University
Outcome results
None listed