A Phase 2 Study of R115777 in Large Granular Lymphocyte (LGL) Leukemia
LGL leukemia is a rare form of cancer that negatively affects either T lymphocyte cells
(T-LGL leukemia) or natural killer cells (NK-LGL leukemia). Both cell types are examples of
white blood cells that originate in the lymph system and bone marrow. T lymphocytes help
fight infection and natural killer cells help attack tumors, both of which are important to
the body's defense system. Individuals with LGL leukemia frequently suffer from low red
blood cell levels (anemia) and low white blood cell levels (neutropenia). Anemic individuals
often feel tired and fatigued because less oxygen is being carried in the blood. Neutropenia
places the body at high risk for developing infections. Currently, individuals with LGL
leukemia are usually treated with immunosuppressive drugs. However, no standard therapy has
been established and some individuals do not respond well to these drugs. Tipifarnib
(R115777), a drug that blocks enzymes needed for the activation of cancer-promoting
proteins, holds potential for treating LGL leukemia. This study will examine the effects of
tipifarnib in individuals diagnosed with T-LGL and NK-LGL leukemia who have anemia and/or
neutropenia.
This study will enroll individuals with a diagnosis of T-LGL leukemia or NK-LGL leukemia. At
an initial screening visit, participants will undergo a physical exam and an
electrocardiogram. Medical histories will be reviewed and blood will be drawn for laboratory
tests. A bone marrow sample will also be taken prior to treatment. Treatment will consist of
a 28-day medication cycle in which participants will receive 300 mg of tipifarnib twice a
day for 21 days, followed by no medication for 7 days. If the initial group of participants
responds favorably to the medication, additional participants will be enrolled into the
study and receive the same treatment dose. If the initial group of participants does not
respond favorably, then additional participants enrolled into the study will receive 600 mg
of tipifarnib. The same 28-day medication dosing schedule will follow.
Study visits will take place once a week. During the first 2 months, blood will be collected
at each visit. Physical exams will occur on a monthly basis throughout the study. After four
months of treatment, each participant's response to the medication will be evaluated.
Participants who have responded well will continue in the study for 1 additional month;
participants who have not responded well will continue in the study for 4 additional months.
All participants will be asked to keep a daily log to record medication use. Follow-up
evaluations will occur every 6 months for 5 years following the end of treatment. At each
follow-up visit, participants will undergo a physical exam and blood collection.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Clinical response to tipifarnib
Measured at Months 4 and 8, and at follow-up evaluations performed twice a year for 5 years following treatment
Yes
Thomas P. Loughran, Jr., MD
Principal Investigator
Penn State University
United States: Food and Drug Administration
RDCRN 5402
NCT00331591
May 2006
May 2007
Name | Location |
---|---|
H. Lee Moffitt Cancer Center | Tampa, Florida 33612 |
The Cleveland Clinic Foundation | Cleveland, Ohio |
Pennsylvania State University | Hershey, Pennsylvania 17033 |