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A Phase I/II Study of RAD001 in Advanced Hepatocellular Carcinoma

Phase 1/Phase 2
18 Years
Open (Enrolling)
Hepatocellular Carcinoma

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Trial Information

A Phase I/II Study of RAD001 in Advanced Hepatocellular Carcinoma

- Participants will be given a supply of the study drug RAD001 to be taken at home. They
will be asked to take the study drug every morning on an empty stomach and will be
given a study drug diary to record the time/date each time they take RAD001. Each 6
week period of time is called a cycle of study treatment.

- We are looking for the highest dose of RAD001 that can be given safely. Therefore not
every participant will receive the same dose of RAD001.

- Participants will come to the clinic every other week. At each of these visits, a
physical examination and blood tests will be performed.

- A CT and MRI will be repeated every 6 weeks during the first 3 cycles of treatment then
every 12 weeks thereafter.

Inclusion Criteria:

- Unresectable of metastatic HCC. Patients must have prior core biopsy to confirm the
diagnosis of HCC and have archived tissues available for correlative studies

- At least one measurable site of disease according to RECIST criteria that has not
been previously irradiated. If it has had previous radiation to teh marker
lesion(s), there must be evidence of progression since the radiation

- 0-2 prior systemic chemotherapy and biologic regimens for hepatocellular carcinoma

- Patients with prior chemoembolization history can participate in the study if the
chemoembolization was performed more than 4 weeks ago and patients must have
measurable disease outside of prior chemoembolization field

- 18 years of age or older

- Minimum of 4 weeks since any major surgery or completion of radiation

- Minimum of 4 weeks since completion of all prior systemic anticancer therapy

- ECOG performance status of 0-2

- CLIP score of equal to or less then 3

- Adequate bone marrow, liver and renal function as outlined in the protocol

Exclusion Criteria:

- Prior treatment with any investigational drug within the preceding 4 weeks

- Chronic treatment with systemic steroids or another immunosuppressive agent

- Uncontrolled brain or leptomeningeal metastases, including patients who continue to
require glucocorticoids for brain or leptomeningeal metastases

- Patients with any severe and/or uncontrolled medical conditions or other condition
that could affect participation in the study

- Known history of HIV seropositivity

- Impairment of gastrointestinal function or gastrointestinal disease that may
significantly alter the absorption of RAD001

- Active, bleeding diathesis

- Women who are pregnant or breast feeding

- Patients who have received prior treatment with an mTor inhibitor

- Patients with known hypersensitivity to RAD001 or other rapamycins or its excipients

- History of non-compliance to medical regimens

- Patients with a positive dipstick for urine protein (reading of 2+ or greater) will
then undergo a 24-hour urine collection for protein. If patients have a 2g or
greater of protein/24hr, they will be excluded from the study.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the maximum tolerated dose of RAD001 in patients with advanced hepatocellular carcinoma (HCC).

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Andrew X. Zhu, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Food and Drug Administration

Study ID:




Start Date:

August 2007

Completion Date:

August 2010

Related Keywords:

  • Hepatocellular Carcinoma
  • liver cancer
  • RAD001
  • Carcinoma
  • Carcinoma, Hepatocellular



Beth Israel Deaconess Medical Center Boston, Massachusetts  02215
Dana-Farber Cancer Institute Boston, Massachusetts  02115
Massachusetts General Hospital Boston, Massachusetts  02114-2617