Phase II Study of Lenalidomide and Darbepoetin Alfa in Myelodysplastic Syndrome (Low to Intermediate-1 Risk Category Excluding 5q Deletion)
Lenalidomide is designed to change the immune system and it may also interfere with the
development of tiny blood vessels that help support tumor growth. Therefore, in theory, it
may decrease or prevent the growth of cancer cells. Darbepoetin alfa is designed to
stimulate production of red blood cells.
If you are found to be eligible to take part in this study, you will take lenalidomide once
a day by mouth on Days 1-21. On Days 22-28, you will not take lenalidomide. Swallow
lenalidomide capsules whole with water at the same time each day. Do not break, chew or open
If you miss a dose of lenalidomide, take it as soon as you remember on the same day. If you
miss taking your dose for the entire day, take your regular dose the next scheduled day (do
NOT take double your regular dose to make up for the missed dose).
Due to the uncertain amount of re-absorption of lenalidomide, there will be no re-dose for
lenalidomide if you vomit the pills.
Every 28 days is called a study "cycle". Since lenalidomide may cause blood clots, you
will take aspirin every day, by mouth, unless your platelet count is too low. Darbepoetin
alfa will be given through a needle under the skin (subcutaneously) once every 2 weeks.
You will be given a drug diary. In this diary, you will record when you take the study
drugs and any side effects you may have.
During Cycle 1, you will have a study visit just before receiving lenalidomide and then once
a month for the first 3 months, then every 3 months after that. At these visits, you will
have a physical exam. Blood (about 2 tablespoons) will be drawn for routine tests. Your drug
diary will be checked and you will return the bottle of the study drug and any unused drug.
You will receive one cycle supply of lenalidomide.
At the end of the first 3 cycles, or earlier if your doctor thinks it is necessary, you
will have a bone marrow aspirate. If your doctor thinks it is necessary, you may have
additional bone marrow aspirates while on study.
You may remain on study for as long as you are benefitting. You will be taken off study if
the disease gets worse or intolerable side effects occur.
Once you are off-study, you will have an end-of-study visit. At this visit, blood (about 2
tablespoons) will be drawn for routine tests. If you are a woman who is able to have
children, a portion of this blood will be used for a blood pregnancy test.
You will be contacted by phone 30 days after you are taken off study, and then every 3
months. You will be asked about any side effects you are experiencing, any new treatments
you are having, and if you have needed any blood products. The phone call should take about
This is an investigational study. Lenalidomide is FDA approved and commercially available.
Lenalidomide is approved for the treatment of patients with transfusion-dependent anemia due
to low- or intermediate-1-risk myelodysplastic syndromes associated with the chromosome 5
abnormality with or without other chromosome abnormalities. Lenalidomide is also approved
in combination with dexamethasone for the treatment of patients with multiple myeloma that
have received at least one prior therapy. Its use in this study is investigational.
Darbepoetin alfa is approved by FDA for treatment of low red blood cell in patients with
cancer and kidney disease. The use of these drugs together is considered to be
investigational. Up to 39 patients will take part in this study. All will be enrolled at
M. D. Anderson.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Patients With a Transfusion Independence Response
Response defined as transfusion independence (no red blood cell transfusions) for at least 8 weeks, anytime during the six 28-day cycles of therapy.
Over six 28-day cycles (approximately 168 days)
Gautam Borthakur, MBBS
UT MD Anderson Cancer Center
United States: Food and Drug Administration
|UT MD Anderson Cancer Center||Houston, Texas 77030|