Clinical trial NCT01954745
Conditions: Bile Duct Cancer Intrahepatic Cholangiocarcinoma Cholangiocarcinoma of the Extrahepatic Bile Duct
Keywords: Bile Duct Cancer intrahepatic cholangiocarcinoma extrahepatic cholangiocarcinoma Cabozantinib
Sponsor: Massachusetts General Hospital
Start date: 2013-09-01; Completion date: 2016-06-01
Enrollment: 19 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This research study is evaluating a drug called cabozantinib as a possible treatment cancer of the bile duct. Cabozantinib is a drug that targets specific pathways inside the cells of the body. By blocking the c-MET and VEGFR2 pathways from sending signals, cabozantinib may prevent cells from multiplying. This drug has been used in other research studies and information from those other research studies suggests that this drug may help to stop the growth of bile duct cancer.

In this research study, the investigators are looking to see how well cabozantinib works in slowing the growth of bile duct cancer. The investigators are also assessing the safety and tolerability of cabozantinib in participants with this type of cancer.

Detailed description:
After the screening procedures confirm that the participants are eligible to participate in the research study:

A two-stage design will be employed in this study where at least 9 of the 20 patients enrolled in the first stage need to be progression-free at 16 weeks before the second stage can proceed. If this criterion is met, an additional 24 patients will be enrolled in the second stage for a total of 44 patients in the study. Patients will be treated with cabozantinib 60 mg daily administered orally continuously for 28-day cycles. Tumor assessments will be performed every 8 weeks until documented disease progression by RECIST criteria or drug intolerance.

In this research study, the participant will be given a study drug-dosing diary for each treatment cycle. Each treatment cycle lasts 28 days (4 weeks), during which time the participant will be taking the study drug, cabozantinib daily. The diary will also include special instructions for taking the study drug(s).

The participant will continue to take cabozantinib for as long as it is tolerated without any unacceptable side effects and the cancer does not get worse.

The following tests and procedures will be done during the research study:

Cycle 1 - On days 1, 8 and 15

* Medical History
* Physical Exam including vital signs and height and weight
* Performance Status
* Review of side effects (day 8 and 15 only)
* Routine blood tests (day 8 and 15 only)
* TSH blood sample on day 1 only

Additional research procedures during Cycle 1:

\- Biomarker blood sample (about 2 teaspoons of blood) on day 3 and day 14

Cycles 2-3 - On days 1 and 15

* Medical History
* Physical Exam including vital signs, height and weight
* Performance Status
* Review of side effects
* EKG - day 1 of each cycle ONLY
* Routine blood tests
* Urine test on Day 1 of each cycle ONLY
* Urine pregnancy test on day 1 of each cycle for women of childbearing potential
* TSH blood sample on day 1 only

Cycle 4 and Beyond: Day 1 ONLY

* Medical History
* Physical Exam including vital signs, height and weight
* Performance Status
* Review of side effects
* EKG
* Routine blood tests
* TSH blood sample on day 1 only
* Urine test
* Urine pregnancy test for women of childbearing potential

The following tests will be done every 2 cycles during the research study:

* CA 19-9 blood sample
* Tumor measurements
* Chest/Abdominal/Pelvic CT/MRI of your tumor

Planned Follow-up:

The participant will return to the clinic between 30-37 days after the last dose of cabozantinib for the following tests and procedures:

* Medical History
* Physical Exam including vital signs, height and weight
* Performance Status
* Review of side effects
* Routine blood tests
* CA 19-9 blood sample
* TSH blood sample

Arms: arm 1: Patients will be treated with cabozantinib 60 mg daily administered orally continuously for 28-day cycles. Tumor assessments will be performed every 8 weeks until documented disease progression by RECIST criteria or drug intolerance.
Interventions: intervention 1: Cabozantinib
Intervention descriptions: intervention 1: Cabozantinib 60 mg (free base weight) per day will be administered daily and continuously for a cycle length of 28 days. Subjects will be provided with a sufficient supply of study treatment and instructions for taking the study treatment on days without scheduled clinic visits. After fasting (with exception of water) for 2 hours, subjects will take study treatment daily with a full glass of water (minimum of 8 oz/ 240 mL) and continue to fast for 1 hour after each dose of study treatment.

Protocol documents: