Phase I Trial of Cabozantinib in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Refractory Acute Myeloid Leukemia Clinical Trial

Official title: Phase I Trial of Cabozantinib in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML).

Status Completed

Clinical Trial Summary

This research study is evaluating a drug called cabozantinib as a possible treatment for acute myeloid leukemia (AML). This research study is a Phase I clinical trial. Phase I trials test the safety of an investigational drug or combination of drugs. Phase I studies also try to define the appropriate dose of the investigational drug to use for further studies. This means that the FDA has not approved giving cabozantinib for use in patients, including patients with your type of cancer.

The study drug cabozantinib works by inhibiting several different proteins which are believed to be involved in the growth and multiplication of the cancerous cells associated with acute myeloid leukemia. This drug has been used in other research studies and information from those other research studies suggests that this drug may help to prevent cancer growth.

The primary purpose of this research study is to determine the highest dose of Cabozantinib that can safely be given without severe or unmanageable side effects. The dose identified in this study will be used in future research studies that seek to determine the role of cabozantinib as a treatment for AML.

Clinical Trial Description

Before the research starts (screening): After signing this consent form, the participant will be asked to undergo some screening tests or procedures to find out if they can be in the research study. Many of these tests and procedures are likely to be part of regular cancer care and may be done even if it turns out that the participant does not take part in the research study. If the participant has had some of these tests or procedures recently, they may or may not have to be repeated.

• A medical history, which includes questions about the participant's past and present treatments, their health history, current medications, blood transfusions, and any allergies the participant, may have.

• Physical exam will be performed which will include the measurement of the following:

weight and height, and vital signs (blood pressure, heart and breathing rate, oral temperature.).

• Performance status, which evaluates how the participant is able to carry on with your usual activities.

• Pregnancy test if the participant is a woman who is able to have children. This will be done with urine or blood.

• Urine test a sample of urine will be taken to check for abnormal cells or signs of infection.

• Thyroid test a blood test to assess for normal functioning of the participant's thyroid gland

• Electrocardiogram (EKG): leads are applied to the participant's chest to record their heart beat and rhythm.

• Echocardiogram (ECHO) or MUGA scan of your heart: An echocardiogram is an ultrasound to see how the participant's heart is functioning. A MUGA is also an ultrasound to see how their heart is functioning. For a MUGA, a radioactive substance is injected into the participant's vein and a picture of their heart is created using a special camera.

• Blood tests: approximately 3-4 teaspoons of blood will be drawn to measure the participant's disease level and to determine if their organs are working well enough to allow them to safely participate in this study.

• Bone marrow aspirate/ biopsy: a needle will be inserted into the participant's hip or breast bone in order to remove a small amount of bone marrow cells. Samples will also be collected for research purposes.

If these tests show that the participant is eligible to participate in the research study, then the participant will begin the study treatment. If the participant does not meet the eligibility criteria, the participant will not be able to participate in this research study.

Additional research procedures to be performed at the time of screening:

• Blood tests: approximately 4 teaspoons of blood will be taken for research purposes. Research samples will be taken and compared later in the study to discover how cabozantinib works in fighting the participant's disease.

• Bone marrow aspirate/ biopsy: Bone marrow aspirate samples, approximately 1 teaspoon, from a bone marrow aspirate/biopsy done prior to screening will be taken for research.

After the screening procedures confirm that the participant is eligible to participate in the research study:

Study Drug (cabozantinib ): If the participant takes part in this research study, the participant will be given a study drug diary for each study cycle. Each study cycle is 28 days (4 weeks). Cabozantinib comes in the form of tablets which the participant will take by mouth once a day. Detailed instructions on how to take cabozantinib can be found in their study drug diary.

Research Study Plan:

Tests and Procedures: The participant will come to the clinic on Days 1, 2, 8, 15, 22, and 28 of cycle 1, days 1, 8, 15, and 28 of cycle 2, and days 1 and 15 of every subsequent cycle. Visits to clinic may be more frequent if determined by the study doctor. The participant will have the following tests and procedures at your visits:

Assessments will be completed while on trial:

• Physical exam

• Performance status

• Urine test

• Blood tests: Both routine and research samples will be drawn throughout the study.

Bone marrow aspirate/ biopsy: A bone marrow aspirate/biopsy will be completed on day 28 of cycle 1 of cabozantinib. A bone marrow biopsy may also be done if the participant's cancer does not appear to be getting better, at approximately day 42 after start of therapy. A bone marrow biopsy/aspirate may also be performed if their disease returns/relapses at any time during this study or to assess for disease response. The bone marrow aspirate/biopsy for this study will not be performed if the participant has withdrawn from the study, or/and the participant has received other treatment for their disease. Bone marrow aspirate samples, approximately one teaspoon, will also be obtained for research at these time points. Please note that a blood sample for research purpose will also be drawn on the day of the participant's bone marrow aspirate/biopsy. Approximately two teaspoons of blood will be drawn. ;

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute
• Refractory Acute Myeloid Leukemia
• Relapsed Acute Myeloid Leukemia

• NCT number: NCT01961765
• Study type: Interventional
• Source: Massachusetts General Hospital
• Status: Completed
• Phase: Phase 1
• Start date: November 2013
• Completion date: January 2017