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Thalidomide in Treating Patients With Myelofibrosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00015821
First received: May 6, 2001
Last updated: January 23, 2013
Last verified: January 2013
History of Changes
  Purpose

Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells


Condition Intervention Phase
Primary Myelofibrosis
 Drug: thalidomide
Other: laboratory biomarker analysis
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)

Resource links provided by NLM:

MedlinePlus related topics: Cancer
Drug Information available for: Thalidomide
U.S. FDA Resources

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart. [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    The proportion of successes will be estimated using the Binomial point estimator (number of successes divided by the total number of evaluable patients) and 95% confidence intervals calculated using the Duffy-Santner algorithm for multi-stage designs.


Secondary Outcome Measures:
  • Survival [ Time Frame: Number of days from registration date to the date of death or last follow-up, assessed up to 5 years ] [ Designated as safety issue: No ]
    Kaplan-Meier survival curves will be generated to estimate survival.

  • Time to progression [ Time Frame: Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years ] [ Designated as safety issue: No ]
    Kaplan-Meier survival curves will be generated to estimate time to progression.

  • Response duration [ Time Frame: Number of days from the first date that objective status = complete or partial response was recorded to the date of disease progression or date of death, whichever comes first, assessed up to 5 years ] [ Designated as safety issue: No ]
    Kaplan-Meier survival curves will be generated to estimate response duration.


Enrollment: 43
Study Start Date: January 2001
Primary Completion Date: April 2003 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment (thalidomide)
Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.
 Drug: thalidomide
Given PO
Other Names:
  • Kevadon
  • Synovir
  • THAL
  • Thalomid
Other: laboratory biomarker analysis
Correlative studies

Detailed Description:

PRIMARY OBJECTIVES:

I. To investigate whether thalidomide, a potent inhibitor of angiogenic and fibrogenic growth factors, is an effective therapeutic agent in patients with MMM. Specifically, to assess whether thalidomide improves anemia and/or organomegaly in patients with MMM.

II. To assess the effects of thalidomide on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF, and their respective receptors.

OUTLINE: This is a multicenter study.

Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.

Patients are followed every 6 months until 5 years from study entry.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Histologically confirmed myelofibrosis with myeloid metaplasia

    • Agnogenic myeloid metaplasia
    • Post-polycythemic myeloid metaplasia
    • Post-thrombocythemic myeloid metaplasia
  • No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis
  • No chromosomal translocation t(9;22) or bcr/abl gene rearrangement
  • Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood
  • Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly
  • Performance status - ECOG 0-2
  • Absolute neutrophil count greater than 750/mm^3
  • Platelet count less than 400,000/mm^3
  • WBC less than 50,000/mm^3
  • Bilirubin no greater than 2 mg/dL (if total bilirubin elevated, direct bilirubin must be normal)
  • AST no greater than 3 times upper limit of normal (ULN)
  • Alkaline phosphatase no greater than 3 times ULN
  • Creatinine no greater than 1.5 mg/dL
  • Creatinine clearance at least 60 mL/min
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile women must use at least 1 highly active method AND 1 additional effective method of contraception for at least 4 weeks before study, during study, and for at least 4 weeks after study
  • Fertile men must use effective contraception during study and for at least 4 weeks after study
  • No uncontrolled infection
  • No concurrent condition that would preclude study
  • No peripheral neuropathy
  • At least 1 month since prior interferon, pirfenidone, anagrelide, or epoetin alfa
  • At least 1 month since prior hydroxyurea or other chemotherapy
  • At least 1 month since prior corticosteroids or androgen derivatives
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00015821

Locations
United States, Minnesota
North Central Cancer Treatment Group
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
Principal Investigator: Michelle Elliott North Central Cancer Treatment Group
  More Information

No publications provided

Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00015821     History of Changes
Other Study ID Numbers: NCI-2012-01853, N9982, U10CA025224, CDR0000068367
Study First Received: May 6, 2001
Last Updated: January 23, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Thalidomide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
 Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents

ClinicalTrials.gov processed this record on May 23, 2013