| General information |
| Database: | DB00132 |
| Objective: | Hepatocellular cancer (hepatocellular carcinoma), as well as cholangiocellular cancer (CCC), has an extremely poor prognosis due to the extent of tumor at diagnosis and the underlying liver disease. Sirolimus is used in the transplantation setting as an immunosuppressive agent, but it also possesses antiproliferative and antiangiogenic properties. The objective of the study was to evaluate the effect of sirolimus on hepatocellular carcinoma and CCC. |
| Authors: | Rizell M, et al |
| Title: | Effects of the mTOR inhibitor sirolimus in patients with hepatocellular and cholangiocellular cancer. |
| Journal: | Int J Clin Oncol |
| Year: | 2008 |
| PMID: | 18307022 |
| Trial Design |
| Clinical Trial Id: | NA |
| Agent: | sirolimus/rapamycin
|
| Target: | Serine/threonineprotein kinase mTOR
|
| Cancer Type: | biliary tract, gallbladder cancer and liver cancer |
| Cancer Subtype: | hepatocellular and cholangiocellular cancer. |
| Therapy Type: | mono |
| Therapeutic Combination Type: | NA |
| Therapeutic Combination Content: | NA |
| Study Type: | a prospective singlearmphase I protocol |
| Key Patients Feature: | patients with hepatocellular and cholangiocellular cancer. |
| Biomarker: | NA |
| Biomark Analysis: | NA |
| Control Group Info: | single arm |
| Treatment Info: | Sirolimus was administered once daily by mouth, with the dose adjusted to a serum trough level between 4 and 15 mug/ml. |
| Primary End Point: | Tumor response was evaluated by CT or MRI, according to RECIST, every third month. |
| Secondary End Point: | overall survival, time to tumor progression, tumor markers, and side effects. |
| Patients Number: | 21 |
| Trial Results |
| DLT_MTD: | NA |
| Objective Response Rate: | NA |
| Disease Control Rate: | Of the patients with hepatocellular carcinoma, one had partial remission (PR) and fi ve patients had stable disease (SD) at 3 months. Of the patients with CCC, three had SD. |
| Median Time to Progression: | NA |
| Median PFS A vs. C: | NA |
| Median OS A vs. C: | The median survival for patients with hepatocellular carcinoma was 6.5 months (range, 0.236 months) and that for patients with CCC was 7 months (range, 2.635 months). |
| Adverse Event(agent arm): | NA |
| Conclusions: | Treatment of hepatocellular carcinoma and CCC with sirolimus can induce temporary PD or SD. This pilot study indicates that sirolimus might be a promising drug for this treatment, but further clinical studies elucidating the biological effects are advocated. |