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A Phase II Single Arm Study to Evaluate the Safety and Efficacy of RAD001 as Monotherapy in Treatment naïve Advanced Cholangiocarcinoma

Phase 2
18 Years
Open (Enrolling)

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Trial Information

A Phase II Single Arm Study to Evaluate the Safety and Efficacy of RAD001 as Monotherapy in Treatment naïve Advanced Cholangiocarcinoma

Cholangiocarcinoma is one of the most common cause of cancer death in Thailand. Patients
with cholangiocarcinoma are often diagnosed at advanced stage. Palliative therapeutic
approaches consisting of percutaneous and endoscopic biliary drainage have usually been used
for these patients, since there is no effective chemotherapeutic treatment for this type of
cancer. Activation of the phosphoinositide-3-kinase (PI3K)/Akt/mTOR signaling pathway is
frequently found in cholangiocarcinoma cells. It has been suggested to be a key step leading
to the progression of cholangiocarcinoma. In this study, the investigators hypothesize that
inhibition of mTOR may be useful in treating cholangiocarcinoma.

Inclusion Criteria:

- Histologic confirmed diagnosis of cholangiocarcinoma.

- Patients must present with disease not amenable to curative surgery.

- ECOG performance status of < 2

- Patients with at least one measurable lesion at baseline as per the RECIST criteria.

- The following laboratory parameters at screening (visit 1):

WBC are equal to or more than 3,000/uL. Platelet are equal to or more than 100,000/uL
Alanine transaminase (ALT) and aspartate transaminase (AST) ≤ 2.5 x ULN. Patients with
known liver metastases: AST and ALT ≤ 5 x ULN Total Bilirubin < 2 mg/dl* (after drainage)
Serum creatinine equal to or less than 2.0 x upper normal limit

- Life expectancy equal to or more than 12 weeks.

- Ability to understand and willingness to sign a written informed consent and to be
able to follow the visit schedule.

- Female patients at child-bearing age must have negative pregnancy test.

- Patients refuse to have treatment with Chemotherapy or Radiation.

Exclusion Criteria:

- Patients within 2 weeks post-minor surgery, 4 weeks post-major surgery to avoid wound
healing complications. Percutaneous biopsies require no waiting time prior to study

- Patients with a recent history of hemoptysis, ≥ 0.5 teaspoon of red blood.

- Patients who have received prior systemic treatment for their metastatic

- Presence of clinically relevant ascites or liver failure.

- Patients with extensive symptomatic fibrosis of the lungs.

- Patients with a known hypersensitivity to RAD001 (everolimus).

- Patients who have previously received mTOR inhibitors (sirolimus, temsirolimus,
everolimus, deforolimus).

- History or clinical evidence of central nervous system (CNS) metastases. Note:
Subjects who have previously-treated CNS metastases (surgery±radiotherapy,
radiosurgery, or gamma knife) and meet all 3 of the following criteria are eligible:

Are asymptomatic Have had no evidence of active CNS metastases for ≥ 6 months prior to
enrollment and Have no requirement for steroids or enzyme-inducing anticonvulsants (EIAC)

- Clinically significant gastrointestinal abnormalities including, but not limited to:

Malabsorption syndrome Major resection of the stomach or small bowel that could affect the
absorption of RAD001 Active peptic ulcer disease Inflammatory bowel disease Ulcerative
colitis, or other gastrointestinal conditions with increased risk of perforation History
of abdominal fistula, gastrointestinal perforation, or intra abdominal abscess within 28
days prior to beginning of study treatment;

- Patients receiving chronic systemic treatment with corticosteroids or another
immunosuppressive agent. Inhaled and topical steroids are acceptable

- Patients with a known history of human immunodeficiency virus seropositivity

- Patients with autoimmune hepatitis

- Patients with an active, bleeding diathesis. Patients may use coumadin or heparin

- Patients who have any severe and/or uncontrolled medical conditions or other
conditions that could affect their participation in the study.

- Patients who have a history of another primary malignancy ≤ 3 years, with the
exception of non-melanoma skin cancer and carcinoma in situ of uterine.

- Female patients who are pregnant or breastfeeding, or adults of reproductive
potential who are not using effective birth control methods.

- Patients who are using other investigational agents or who had received
investigational drugs ≤ 4 weeks prior to study treatment start.

- Patients unwilling or unable to comply with the protocol.

- Patient who will have got benefit from Radiation or Chemotherapy.

Type of Study:


Study Design:

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

Outcome Measure:

progression free survival in cholangiocarcinoma patients whom treated with everolimus

Outcome Time Frame:

One year

Safety Issue:


Principal Investigator

Kawin Leelawat, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Rajavithi Hospital


Thailand: Ministry of Public Health

Study ID:




Start Date:

January 2012

Completion Date:

June 2014

Related Keywords:

  • Cholangiocarcinoma
  • cholangiocarcinoma
  • RAD001
  • everolimus
  • Cholangiocarcinoma