RAD001 in Advanced Hepatocellular Carcinoma

NCT00516165 | Completed Phase 1 Results DMC

• 1 other identifier: 06-352
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Hepatocellular Carcinoma

Keywords

liver cancer; RAD001

Outcome Measures

Primary Outcomes (2)

• Maximum Tolerated Dose of RAD001 in Patients With Advanced Hepatocellular Carcinoma (HCC).

  2 years

• Progression-free Survival Rate at 24 Weeks

  Progression was defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions" This information will be collected during two years of patient participation.

  2 years

Secondary Outcomes (4)

• Number of Patients With Adverse Events Who Were Treated With RAD001 for Advanced HCC

  2 years

• Overall Response Rate

  2 years

• Time to Progression

  2 years

• Overall Survival

  2 years

Study Arms (1)

RAD001

EXPERIMENTAL

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

Interventions

RAD001 DRUG

Oral pills taken daily in a 42-day cycle (6 weeks). Cycles will be repeated every 42 days

Also known as: Everolimus

RAD001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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
• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Dana-Farber Cancer Institute: collaborator
• Beth Israel Deaconess Medical Center: collaborator
• Novartis: collaborator

Study Sites (3)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Related Publications (1)

• Treiber G. mTOR inhibitors for hepatocellular cancer: a forward-moving target. Expert Rev Anticancer Ther. 2009 Feb;9(2):247-61. doi: 10.1586/14737140.9.2.247.

  PMID: 19192962 DERIVED

MeSH Terms

Conditions

Carcinoma, Hepatocellular; Liver Neoplasms

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Results Point of Contact

• Title: Dr.Andrew X. Zhu
• Organization: Massachusetts General Hospital

azhu@mgh.harvard.edu

617-724-4000

Study Officials

• Andrew X. Zhu, MD, PhD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 13, 2007
• First Posted: August 15, 2007
• Study Start: August 1, 2007
• Primary Completion: January 1, 2010
• Study Completion: November 1, 2011
• Last Updated: February 7, 2017
• Results First Posted: February 7, 2017

Record last verified: 2016-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)