Phase II Study of CC-5013 in Myelofibrosis
Lenalidomide blocks the activity of a substance in the blood called tumor necrosis factor
alpha. Tumor necrosis factor alpha is a substance that is believed to prevent new blood
cells from forming in the bone marrow. Lenalidomide is also believed to help the body's
immune system fight diseases.
Before treatment starts, you will have a complete physical exam, including blood (about 3
teaspoonfuls) and urine tests. A bone marrow sample will be taken. To collect a bone marrow
sample, an area of the hip or chest bone is numbed with anesthetic and a small amount of
bone marrow is withdrawn through a large needle. An ECG (test to measure the electrical
activity of the heart) may be performed.
Women who are able to have children must have a negative pregnancy test [blood (about 1
teaspoon) or urine]. These pregnancy tests must occur within 10 - 14 days and again within
24 hours before the start of lenalidomide. Women who are able to have children with regular
or no menstruation must have a pregnancy test weekly for the first 28 days and then every 28
days while on therapy (including breaks in therapy); when they stop taking lenalidomide and
at Day 28 after the last dose of lenalidomide. Females with irregular menstruation must
have a pregnancy test weekly for the first 28 days and then every 14 days while on therapy
(including breaks in therapy), when they stop taking lenalidomide and at Day 14 and Day 28
after the last dose of lenalidomide.
You are considered to be a woman who is able to have children if you are a sexually mature
female who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not
been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at
any time in the preceding 24 consecutive months).
You will take 2 lenalidomide capsules by mouth daily. You should swallow lenalidomide
capsules whole with water at the same time each day. Do not break, chew or open the
capsules. 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).
If your platelet count is less than 100,000 at the time of study enrollment, the dose will
be one capsule daily. The dose may be decreased depending on side effects. The dose may be
increased if needed to better control the disease. This will be decided cycle by cycle.
During treatment, you will give blood samples (about 1 tablespoon each) about every week.
The tests may be repeated more frequently to check for side effects. You will need to
return to M. D. Anderson monthly for the first 3 months, then at least every 3 months
afterwards (while on the study) in order to be evaluated for response and tolerance to
lenalidomide. Only one 28-day cycle of lenalidomide may be given to you for each cycle per
You may continue to receive this therapy as long as there are no severe side effects or
worsening of the disease. You will be asked to keep diaries documenting when you take the
capsules. You will also need to return empty medication bottles at each visit. If you have
had 4 to 6 months of treatment without any evidence of benefit, you may be taken off the
This is an investigational study. Lenalidomide is a new drug related to the drug called
thalidomide. Lenalidomide is approved by the Food and Drug Administration (FDA) for the
treatment of specific types of myelodysplastic syndrome (MDS) and in combination with
dexamethasone for patients with multiple myeloma (MM) who have received at least 1 prior
therapy. MDS and MM are cancers of the blood. It is currently being tested in a variety of
cancer conditions. In this case it is considered investigational. Up to 41 participants may
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
Efficacy of CC-5013 in Myelofibrosis
Response evaluation, sustained for 2 weeks: Complete Remission (Neutrophil count between 1 to 10 x 10^9/L without peripheral blasts in blood or bone marrow); Partial Hematologic Response/Partial Remission (Increase in neutrophil by 50% + above 10^9/L for neutropenia); Hematologic Improvement (increase in Neutrophil count, hemoglobin, platelet count or reduction in blood/marrow blasts) or No Response. If nine or < patients respond to therapy (response other than 'No Response'), therapy declared ineffective. However, if 11 or > patients respond to therapy, therapy considered efficacious.
3 - 4 Months for all patients; 24 months for responders
Jorge E Cortes, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
|M.D. Anderson Cancer Center||Houston, Texas 77030|