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Trial record 22 of 50 for:    "Essential Thrombocythemia" | "Anti-Infective Agents"

Thalidomide, Prednisone, and Cyclophosphamide in Treating Patients With Myelofibrosis and Myeloid Metaplasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00445900
Recruitment Status : Completed
First Posted : March 9, 2007
Last Update Posted : March 17, 2011
National Cancer Institute (NCI)
Information provided by:
Mayo Clinic

Brief Summary:

RATIONALE: Giving thalidomide together with prednisone and cyclophosphamide may lessen symptoms caused by myelofibrosis and myeloid metaplasia.

PURPOSE: This phase II trial is studying the side effects and how well giving thalidomide together with prednisone and cyclophosphamide works in treating patients with myelofibrosis and myeloid metaplasia.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Secondary Myelofibrosis Drug: cyclophosphamide Drug: prednisone Drug: thalidomide Other: immunohistochemistry staining method Other: laboratory biomarker analysis Procedure: biopsy Phase 2

Detailed Description:



  • Determine the benefit of thalidomide, prednisone, and cyclophosphamide in alleviating disease-associated anemia, thrombocytopenia, and/or splenomegaly in patients with myelofibrosis with myeloid metaplasia (MMM).
  • Determine the benefit of this regimen in palliating four hypercatabolic constitutional symptoms (i.e., weight loss, fatigue, drenching night sweats, and unexplained fevers) in these patients.
  • Determine the toxicity profile of this regimen in these patients.


  • Determine the effect of this regimen on leukocyte count.
  • Determine the effect of this regimen on bone marrow histology, including microvessel density and reticulin fibrosis.
  • Determine the effect of this regimen on intramedullary and urinary markers of angiogenesis.
  • Determine the effect of this regimen on circulating myeloid progenitor cells by quantifying CD34+ cells.

OUTLINE: Patients receive oral thalidomide, oral prednisone, and oral cyclophosphamide (TPC) once daily on days 1-28. Treatment repeats every 28 days for 3 courses. After 3 courses (3 months) of treatment, patients who respond to TPC therapy may receive oral thalidomide alone once daily for up to 3 months in the absence of disease progression or unacceptable toxicity.

Patients undergo bone marrow aspirate and biopsy prior to study entry, 6 months after starting therapy, and then every 6 months for up to 3 years. Samples are analyzed by microvessel density/angiogenesis studies (i.e., CD34 immunohistochemical and vascular endothelium-specific staining) to determine the effect of therapy on markers of bone marrow angiogenesis.

After completion of study therapy, patients are followed every 6 months for up to 3 years.

PROJECTED ACCRUAL: A total of 22 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of the Combination of Low-Dose Thalidomide, Prednisone, and Oral Cyclophosphamide ("TPC") in the Therapy of Myelofibrosis With Myeloid Metaplasia (MMM)
Study Start Date : October 2004
Actual Primary Completion Date : October 2006
Actual Study Completion Date : October 2006

Primary Outcome Measures :
  1. Confirmed response, defined as a complete or partial response in ≥ 1 of 3 response categories (i.e., anemia, thrombocytopenia, or splenomegaly or hepatomegaly)

Secondary Outcome Measures :
  1. Constitutional symptom status and bone marrow morphology
  2. Overall survival
  3. Progression-free survival
  4. Time to progression
  5. Duration of response
  6. Toxicity as measured by NCI CTC v 2.0

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed myelofibrosis with myeloid metaplasia (MMM) of any of the following subtypes:

    • Agnogenic myeloid metaplasia
    • Post-polycythemic myeloid metaplasia
    • Post-thrombocythemic myeloid metaplasia
  • Must have 1 of the following MMM-related conditions:

    • Anemia, defined as hemoglobin < 10 g/dL

      • Iron deficiency must be excluded as cause
    • Thrombocytopenia, defined as platelet count < 100,000/mm³
    • Palpable hepatomegaly or splenomegaly
  • No evidence of myelofibrosis-associated conditions in the bone marrow, including any of the following:

    • Metastatic carcinoma
    • Lymphoma
    • Myelodysplasia
    • Hairy cell leukemia
    • Mast cell disease
    • Acute leukemia (including M7 type)
    • Acute myelofibrosis
  • No chromosomal translocation t(9:22) or bcr-abl as determined by bone marrow chromosome analysis or peripheral blood fluorescent in situ hybridization (FISH) analysis


  • ECOG performance status 0-3
  • Absolute neutrophil count ≥ 750/mm³
  • Bilirubin ≤ 2 times upper limit of normal (ULN), unless elevation due to MMM
  • AST ≤ 5 times ULN, unless elevation due to MMM
  • Creatinine ≤ 2.5 mg/dL
  • No uncontrolled infection, including tuberculosis

    • No known history of positive purified protein derivative (PPD) untreated by isoniazid therapy

      • Positive PPD with normal chest X-ray and completion of full-course isoniazid therapy allowed
  • No federal medical center inmates or other incarcerated patients
  • No peripheral neuropathy ≥ grade 2
  • No comorbid condition in which the use of study therapy is felt to be potentially harmful
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use 2 forms of effective contraception


  • No chemotherapy (e.g., hydroxyurea, myelosuppressive therapy) within the past 14 days
  • Prior splenectomy for MMM allowed
  • No concurrent hematopoietic growth factors

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00445900

Sponsors and Collaborators
Mayo Clinic
National Cancer Institute (NCI)
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Study Chair: Ruben A. Mesa, MD Mayo Clinic

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Responsible Party: Ruben A. Mesa, M.D., Mayo Clinic Identifier: NCT00445900     History of Changes
Other Study ID Numbers: CDR0000530973
P30CA015083 ( U.S. NIH Grant/Contract )
MC028A ( Other Identifier: Mayo Clinic Cancer Center )
1360-03 ( Other Identifier: Mayo Clinic IRB )
First Posted: March 9, 2007    Key Record Dates
Last Update Posted: March 17, 2011
Last Verified: March 2011
Keywords provided by Mayo Clinic:
primary myelofibrosis
essential thrombocythemia
polycythemia vera
secondary myelofibrosis
Additional relevant MeSH terms:
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Anti-Infective Agents
Primary Myelofibrosis
Myeloproliferative Disorders
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Leprostatic Agents
Anti-Bacterial Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors