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RAD001 in Advanced Hepatocellular Carcinoma

This study has been completed.
Sponsor:
Collaborators:
Dana-Farber Cancer Institute
Beth Israel Deaconess Medical Center
Novartis
Information provided by (Responsible Party):
Andrew X. Zhu, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT00516165
First received: August 13, 2007
Last updated: March 16, 2014
Last verified: March 2014
  Purpose

Laboratory studies have shown that RAD001 can prevent cells from multiplying. Consequently, the study drug is being tested in medical conditions in which excessive cell multiplication (as in cancer) needs to be stopped. The main purpose of this research study is to find the highest dose of RAD001 that can be given safely (without causing severe side effects) and to learn the effects (good or bad) RAD001 has on participants with liver cancer.


Condition Intervention Phase
Hepatocellular Carcinoma
Drug: RAD001
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Study of RAD001 in Advanced Hepatocellular Carcinoma

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Maximum tolerated dose of RAD001 in patients with advanced hepatocellular carcinoma (HCC). [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
  • Progression-free survival rate at 24 weeks [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of patients with adverse events who were treated with RAD001 for advanced HCC [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
  • Overall response rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Time to progression [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Overall survival [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Enrollment: 28
Study Start Date: August 2007
Study Completion Date: November 2011
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAD001
Patients will receive RAD001 10 mg/day orally (6 weeks/cycle). Patients will be continued on treatment until disease progression, limiting toxicity, patient withdrawal of consent, or death.
Drug: RAD001
Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days
Other Name: Everolimus

Detailed Description:
  • 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.
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00516165

Locations
United States, Massachusetts
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
Dana-Farber Cancer Institute
Beth Israel Deaconess Medical Center
Novartis
Investigators
Principal Investigator: Andrew X. Zhu, MD, PhD Massachusetts General Hospital
  More Information

No publications provided by Massachusetts General Hospital

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Andrew X. Zhu, MD, Principal Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00516165     History of Changes
Other Study ID Numbers: 06-352
Study First Received: August 13, 2007
Last Updated: March 16, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Massachusetts General Hospital:
liver cancer
RAD001

Additional relevant MeSH terms:
Carcinoma
Carcinoma, Hepatocellular
Adenocarcinoma
Digestive System Diseases
Digestive System Neoplasms
Liver Diseases
Liver Neoplasms
Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Glandular and Epithelial
Everolimus
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 27, 2014