A Phase I Open-label Study to Assess the Safety, Tolerability and Preliminary Efficacy of BKM120, PI3K Kinase Inhibitor, in Patients With Advanced Leukemias
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.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose of BKM 120
Maximum tolerated dose (MTD) defined as highest dose in which 1/6 or less subjects experience a dose limiting toxicity (DLT) during the first course of treatment.
28 days, Cycle 1
Yes
Marina Konopleva, MD, PHD
Principal Investigator
UT MD Anderson Cancer Center
United States: Food and Drug Administration
2010-0874
NCT01396499
July 2012
Name | Location |
---|---|
UT MD Anderson Cancer Center | Houston, Texas 77030 |