Study to Assess Safety, Tolerability and Preliminary Efficacy of BKM120, PI3K Kinase Inhibitor, With Advanced Leukemias

Leukemia Clinical Trial

Official title: A Phase I Open-label Study to Assess the Safety, Tolerability and Preliminary Efficacy of BKM120, PI3K Kinase Inhibitor, in Patients With Advanced Leukemias

Status Completed

Clinical Trial Summary

The goal of this clinical research study is to find the highest tolerable dose of BKM120 that can be given to patients with relapsed or refractory leukemia. The safety of BKM120 will also be studied.

Clinical Trial Description

The Study Drug:

BKM120 is designed to block a protein that is important to the growth and division of cancer cells, which may cause the cells to die.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will be assigned to a dose level of BKM120 based on when you join this study. Up to 2 dose levels of BKM120 will be tested. Up to six (6) participants will be enrolled at each dose level. The first group of participants will receive the lower dose level.

You will take BKM120 tablets by mouth with a large glass of water every day you are on this study. You should take BKM120 about 1 hour after a light breakfast. You should take BKM120 at about the same time each day. You need to fast (not eat or drink anything except water) for 2 hours after you take your BKM120 dose.

You should swallow the BKM120 tablets whole. The tablets must not be chewed, broken, or crushed. If you vomit after taking BKM120, you should not take another tablet that day to make up for that dose. If you forget to take BKM120 one morning, call your doctor and ask for instructions. Do not take BKM120 after 6pm of the same day or take any extra doses to make up for the missed dose.

If your study doctor feels it is needed or if you have side effects, BKM120 may be stopped and then started again at a lower dose or may be stopped completely.

You will be asked to record in a pill diary each dose you take. You will return the pill diary, unused study drug, and pill containers to the clinic staff at the end of each cycle.

Study Visits:

On this study, each study cycle will last 28 days. You will have study visits every other week (Days 1 and 15) during the first 2 cycles of the study, and then once a month after that. The following tests and procedures will be performed at all study visits:

• Your medical history will be recorded

• You will have a physical exam, including measurement of your vital signs.

• Your performance status will be recorded.

• You will be asked about any drugs you may be taking and about any side effects you may be having.

• Blood (about 1 tablespoon) will be drawn for routine tests and to check your blood sugar. ° You will need to fast for 8 hours before this blood draw.

• You will complete the questionnaires about your mood.

On Day 28 of Cycle 1 and every 3 months after that, you will have a bone marrow aspirate and/or biopsy to check the status of the disease. You may have additional bone marrow aspirations collected while you are on study if the doctor thinks they are needed.

Every 4 months, you will have a MUGA scan or an ECHO to check the status of the disease.

ECGs and chest x-rays may be performed throughout the study to check your heart and lung function if the doctor thinks they are needed.

Length of Study:

You may continue taking the study drug for as long as 12 cycles if the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Your participation on the study will be over once you have completed the end-of-study visit.

End-of-Study Visit:

After you stop taking BKM120, you will have an end-of-study visit. The following tests and procedures will be performed:

• You will have a physical exam, including measurement of your vital signs.

• Your performance status will be recorded.

• You will be asked about any drugs you may be currently taking or have taken.

• You will complete the questionnaires about your mood.

• Blood (about 1 tablespoon) and urine will be collected for routine tests. The blood will also be used to check your kidney and liver function and to check your blood sugar. You will need to fast for 8 hours before this blood draw.

• You will have a MUGA scan or an ECHO to check the status of the disease.

• You will have a bone marrow aspirate and/or biopsy to check the status of the disease.

This is an investigational study. BKM120 is not FDA approved or commercially available. It is currently being used for research purposes only.

Up to 16 patients take part in this study. All will be enrolled at MD Anderson. ;

Study Design

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

Related Conditions & MeSH terms

• Leukemia

• NCT number: NCT01396499
• Study type: Interventional
• Source: M.D. Anderson Cancer Center
• Status: Completed
• Phase: Phase 1
• Start date: July 2012
• Completion date: October 2016