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A Phase II, Prospective, Open Label Study to Determine the Safety and Efficacy of Pomalidomide (CC-4047) in Subjects With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF; Post-PV MF, or Post-ET MF)


Phase 2
18 Years
N/A
Open (Enrolling)
Both
Polycythemia Vera, Thrombocythemia

Thank you

Trial Information

A Phase II, Prospective, Open Label Study to Determine the Safety and Efficacy of Pomalidomide (CC-4047) in Subjects With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF; Post-PV MF, or Post-ET MF)


The Study Drug:

CC-4047 is a drug that may also affect the growth of blood vessels that support tumor
growth. If these blood vessels stop growing, it may stop cancer cell growth.

Prednisone is a corticosteroid that is similar to a natural hormone made by your body.
Prednisone is often given in combination with other chemotherapy to treat cancer.

Study Drug Administration:

On Days 1- 28 of every 28-day study "cycle," you will take CC-4047 capsule(s) by mouth. You
should take the capsule(s), about the same time every day. The capsule(s) should not be
opened, broken, or chewed.

You will receive prednisone by mouth during the first 3 cycles of therapy. You will take it
1 time a day during Cycle 1. During Cycle 2, you will take a smaller dose 1 time a day.
During Cycle 3, you will take the same dose as in Cycle 2, but only 1 time every other day.

On Days 1-28, you will also take low-dose aspirin. This should be taken at the same time as
CC-4047. Aspirin is take to help prevent blood clots, which may occur from taking CC-4047.
If you are unable to take aspirin, your study doctor will have you take another drug.

You will be given a study "diary". In this diary, you will record when you take each dose
of the study drug. You will return any unused study drug and empty bottles at each visit.

Study Visits:

On Day 1 of Cycles 1 and 2, the following tests and procedures will be performed:

- You will have a physical exam, including measurements of your vital signs (blood
pressure, temperature, and heart rate) and weight.

- You will be asked about any drugs you are taking and about recent blood transfusions
you may have had.

- You will have a performance status evaluation.

- Blood (about 1 tablespoon) will be drawn for routine tests.

On Days 1, 8, 15, and 22 of Cycles 1 and 2, blood (about 4 teaspoons) will be drawn for
routine tests.

On Day 28 of each cycle, the following tests and procedures will be performed:

- You will be asked about any drugs you are taking and about recent blood transfusions
you may have had.

- You will be asked about any birth control methods you are using.

- You will have a physical exam, including measurement of your vital signs and weight.

- Measurements of your spleen and/or liver will be taken.

- You will have a performance status evaluation.

- Blood (about 4 tablespoons) will be drawn for routine tests.

- You will be asked about any new side effects or medical conditions you may have
experienced.

- You will complete a questionnaire about your quality-of-life as well as an assessment
of possible pain in your spleen. (Cycles 1 and 2 only)

On Day 28 of every third cycle (Cycle 3, 6, 9, and so on), the following tests and
procedures will be performed:

- Blood (about 4 teaspoons) will be drawn to test you thyroid function.

- You will have an ECG.

- Response of disease to therapy will be assessed. Bone marrow biopsy and aspirate will
be done to confirm a complete response if your doctor thinks it is needed.

Pregnancy Testing:

Women who are able to have children will have a pregnancy test every week during Cycle 1,
and then every 28 days after that if your menstrual cycle is regular (If your menstrual
cycle is not regular, you will have a blood pregnancy test every 2 weeks). These pregnancy
tests will be part of a routine blood draw.

Length of Study:

You will remain on study treatment as long as the therapy is beneficial to you. You will be
taken off study early if the disease gets worse or intolerable side effects occur.

End-of-Treatment Visit:

Once you are off study, you will have an end-of-study visit. At this visit, the following
tests and procedures will be performed:

- You will have a physical exam, including measurements of your vital signs and
measurements of your spleen and/or liver.

- You will have an ECG.

- You will be asked about any drugs you are taking and about any blood transfusions you
may have had.

- Blood (about 4 tablespoons) and urine will be collected for routine tests and thyroid
function tests. This routine blood draw will include a pregnancy test for women who
are able to have children.

- You will be asked about any side effects you may have experienced.

Follow-Up Visit:

Women who are able to have children will have a blood (about 1 tablespoon) pregnancy test 28
days after the last dose of study drug. If your periods are irregular you will have 2 blood
(about 1 tablespoon each time) pregnancy tests, one 14 days after the last dose of study
drug and another 28 days after the last dose of study drug. Response of disease to therapy
will be assessed at follow-up 28 days after last dose of study drug.

This is an investigational study. CC-4047 (pomalidomide) is FDA approved and commercially
available for the treatment of certain types of MM. Its use in this study is
investigational.

Prednisone is FDA approved and commercially available.

Up to 70 patients will take part in this study. All will be enrolled at M. D. Anderson.


Inclusion Criteria:



1. Must be >/= 18 years of age at the time of voluntarily signing an Institutional
Review Board/Independent Ethics Committee (IRB/IEC) - approved informed consent form.

2. Must be diagnosed with myelofibrosis requiring therapy including myelofibrosis with
myeloid metaplasia (MMM), de novo presentation (i.e. agnogenic myeloid metaplasia
[AMMM], and developing after an antecedent history of Polycythemia vera (i.e.,
post-polycythemic myeloid metaplasia [PPMM]), or essential Polycythemia (i.e., post
thrombocythemic myeloid metaplasia [PTMM]).

3. Screening total hemoglobin level < 10 g/dL or transfusion-dependent anemia defined as
per IWG criteria (transfusion dependency defined by a history of a least 2 units of
red blood cell transfusions in the last 28 days for hemoglobin < 8.5 g/dL that was
not associated with overt bleeding)

4. Must have adequate organ function as demonstrated by the following to starting study drug: ·ALT (SGOT) and AST (SGPT) (ULN), [unless upon judgment of the treating physician, it is believed to be due to
extramedullary hematopoiesis (EMH)] ·Total bilirubin < 3 x ULN or Direct Bilirubin <
2 x ULN ·Serum creatinine /= 1,000/µL
(>/=1.0 x 10^9/L) ·Platelet count >/= 50,000/µL (>/=50 x 10^9/L)

5. Subjects must be willing to receive transfusion of blood products

6. ECOG performance status (PS) of 0, 1, or 2 at screening.

7. Must be willing to adhere to the study visit schedule and other protocol
requirements.

8. No active malignancies with the exception of basal cell or squamous cell carcinoma of
the skin, or carcinoma (in situ) of the cervix or breast

9. Females of childbearing potential (FCBP) must agree to use two reliable forms of
contraception simultaneously or abstinence from intercourse during the following time
periods: At least 28 days before starting study drug While participating in the study
For at least 28 days after discontinuation from the study. Two methods of reliable
contraception must include one highly effective method, hormonal pills, injections,
or implants, tubal ligation, partner's vasectomy and one additional effective method
(i.e. latex condom, diaphragm, cervical cap)

Exclusion Criteria:

1. Known positive status for HIV, hepatitis B carrier, or active hepatitis C infection.

2. The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea),
corticosteroids, or experimental drug or therapy within 14 days of starting CC-4047
and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.

3. Any serious medical condition or psychiatric illness that would prevent, (as judged
by the treating physician) the subject from signing the informed consent form or any
condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study.

4. Pregnant or lactating females

5. Prior use of CC-4047

6. Currently enrolled on another clinical trial or receiving investigational agent

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

Number of Patients with Response

Outcome Time Frame:

6 months

Safety Issue:

No

Principal Investigator

Srdan Verstovsek, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

2007-0199

NCT ID:

NCT00946270

Start Date:

July 2009

Completion Date:

Related Keywords:

  • Polycythemia Vera
  • Thrombocythemia
  • CC-4047
  • Pomalidomide
  • polycythemia vera
  • essential thrombocythemia myelofibrosis
  • Primary Myelofibrosis
  • Polycythemia
  • Polycythemia Vera
  • Thrombocythemia, Essential
  • Thrombocytosis

Name

Location

UT MD Anderson Cancer CenterHouston, Texas  77030