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A Phase I/II, Prospective, Open-Label Study to Determine the Safety and Efficacy of CC-4047 in Patients With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis®


Phase 1/Phase 2
18 Years
N/A
Open (Enrolling)
Both
Chronic Myeloproliferative Disorders, Secondary Myelofibrosis

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Trial Information

A Phase I/II, Prospective, Open-Label Study to Determine the Safety and Efficacy of CC-4047 in Patients With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis®


OBJECTIVES:

Primary

- To determine the maximum tolerated dose of CC-4047 in patients with primary
myelofibrosis and post polycythemia vera or post essential thrombocythemia
myelofibrosis. (Phase I [closed to accrual as of 12/3/2008])

- To determine the effect of this drug in these patients. (Phase II [open to accrual as
of 1/7/2009])

- To determine the safety of this drug in these patients. (Phase II [open to accrual as
of 1/7/2009])

Secondary

- To further evaluate the nature and quality of responses to CC-4047.

OUTLINE: This is a phase I dose-escalation study followed by a phase II study.

- Phase I (closed to accrual as of 12/3/2008): Patients receive oral CC-4047 on days
1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease
progression or unacceptable toxicity. After 12 courses, patients with responding
disease may continue to receive CC-4047 in the absence of disease progression or
unacceptable toxicity.

- Phase II (open to accrual as of 1/7/2009): Patients receive oral CC-4047 at the maximum
tolerated dose determined in phase I.

Patients complete quality of life questionnaires at baseline, every 28 days for the first 3
courses, and then every 3 courses (every 84 days) thereafter.

After completion of study treatment, patients are followed at 28 days and then every 6
months for up to 3 years.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of primary and post essential thrombocythemia (ET) or post polycythemia
vera (PV) myelofibrosis requiring therapy

- De novo presentation (i.e., agnogenic myeloid metaplasia AND post ET or post PV
myelofibrosis)

- Developed after an antecedent history of PV (i.e., post polycythemic myeloid
metaplasia) or essential polycythemia (i.e., post thrombocythemic myeloid
metaplasia)

- Total hemoglobin < 10 g/dL OR transfusion dependent anemia (defined by a history of ≥
2 units of RBC transfusions within the past 28 days for hemoglobin < 8.5 g/dL that
was not associated with overt bleeding) OR marked splenomegaly (e.g., ≥ 10 cm below
costal margin)

PATIENT CHARACTERISTICS:

- ECOG performance status 0-2

- ANC ≥ 500/μL

- Platelet count ≥ 20,000/μL

- AST and ALT ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if attributed to
hepatic extramedullary hematopoiesis)

- Total bilirubin ≤ 3 times ULN OR direct bilirubin ≤ 2 times ULN

- Serum creatinine ≤ 2.0 mg/dL

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective double-method contraception for ≥ 28 days before,
during, and for ≥ 28 days after completion of study treatment

- Agrees to abstain from donating blood, semen, or sperm during and for ≥ 28 days after
completion of study treatment

- Willing to undergo transfusion of blood products (if applicable)

- Able to complete questionnaire(s) alone or with assistance

- No known HIV positivity, hepatitis B carrier, or active hepatitis C infection

- No serious medical condition, psychiatric illness, or any other condition, including
the presence of laboratory abnormalities, that (as judged by the treating physician)
would preclude giving informed consent or participating in the study or confound the
ability to interpret data from the study

- No other active malignancies, except basal cell or squamous cell carcinoma of the
skin or carcinoma in situ of the cervix or breast

- No active deep vein thrombosis or pulmonary embolism that has not been
therapeutically anticoagulated

PRIOR CONCURRENT THERAPY:

- Recovered from all prior therapy

- No prior CC-4047

- More than 28 days since prior growth factors, cytotoxic chemotherapeutic agents
(e.g., hydroxyurea or anagrelide), corticosteroids, or experimental drugs or
therapies

- No other concurrent experimental drugs or therapies or cytotoxic chemotherapeutic
agents (e.g., hydroxyurea or anagrelide) for myelofibrosis

- No concurrent growth factors (including erythropoietin) for myelofibrosis, except
G-CSF or pegfilgrastim

- No concurrent chronic use (i.e., > 2 weeks) of more than physiologic doses of
corticosteroids (dose equivalent to > 10 mg/day of prednisone)

Type of Study:

Interventional

Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Rate and frequency of dose-limiting toxicity as measured by NCI CTCAE v3.0

Safety Issue:

Yes

Principal Investigator

Ruben A. Mesa, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic

Authority:

United States: Food and Drug Administration

Study ID:

MC078B

NCT ID:

NCT00669578

Start Date:

May 2008

Completion Date:

May 2013

Related Keywords:

  • Chronic Myeloproliferative Disorders
  • Secondary Myelofibrosis
  • secondary myelofibrosis
  • polycythemia vera
  • essential thrombocythemia
  • primary myelofibrosis
  • Primary Myelofibrosis
  • Myeloproliferative Disorders
  • Polycythemia
  • Polycythemia Vera
  • Thrombocythemia, Essential
  • Thrombocytosis

Name

Location

Mayo ClinicRochester, Minnesota  55905