TSC-associated renal angiomyolipoma
Conditions
Interventions
Sponsors
Peking Union Medical College Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 85 Years
Inclusion criteria
Inclusion criteria: 1. Men or women (not pregnant) older than 18 years; 2. Clinical diagnosis of TSC-AML; 3. CT or MRI shows one or more TSC-AMLs with the maximum diameter more than at least 3 cm; 4. Without AML bleeding or embolism in the past 6 months.
Exclusion criteria
Exclusion criteria: 1. Women who plan to be pregnant or have been pregnant or lactating; 2. CT or MRI shows the maximum diameter of TSC-AML less than 3 cm; 3.Patients expected to undergo surgery or embolization therapy during the trial; 4.History of coronary heart disease, myocardial infarction or cerebral infarction related to atherosclerosis; 5. History of AML bleeding or embolism in the past 6 months; 6. Impaired lung function defined as following: (1) For patients without lymphangioleiomyomatosis (LAM) -Known impaired lung function (e.g. FEV1 or DLco less than 70% of predicted) (2) For patients with LAM; -DLco less than 35%, or -O2 saturations below normal at rest, or -O2 saturation less than at least 88% on 6 minute walking test with up to 6 liters O2/minute nasal oxygen 7. Severe hematological disease or liver function abnormality (such as aminotransferase more than 2.5 times of normal upper limit, serum bilirubin more than 1.5 times of normal upper limit, hemoglobin more than 9 g/dL, platelet more than 80,000/mm3, or absolute neutrophil count less than 1000/mm3); 8. Concomitant severe infection before or during the trial; 9. Previous organ transplantation; 10. History of other surgeries (involving entry into body cavity or suture) in the past 2 months; 11. Previous mTOR inhibitor treatment (such as rapamycin, sirolimus and everolimus); 12. Use of investigational drug within 30 days; 13. Poor control of hyperlipidemia: fasting serum cholesterol more than 300 mg/dL (or more than 7.75 mmol/L), fasting triglyceride more than 2.5 times of normal upper limit; 14. Poor control of diabetes: fasting blood glucose more than 1.5 times of normal upper limit; 15. Patients with bleeding tendency or using oral anti-vitamin K drugs (except low-dose warfarin); 16. History of HIV seropositivity; 17. Active hepatitis; 18. Patients who cannot participate in regular visits and follow-up; 19. Patients who are not suitable for MRI examination (such as excessive obesity, mental disorders, bullet fragment in body, stent and pacemaker); 20. Serum creatinine more than 1.5 times of normal upper limit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Maximum tumor diameter or volume;mutations of TSC1 and TSC2;VEGF; | — |
Countries
China
Contacts
Public ContactLi Han-zhong, Zhang Yu-shi
Outcome results
None listed