Thalidomide in Treating Patients With Myelofibrosis

October 7, 2013 updated by: National Cancer Institute (NCI)

A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)

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.

Study Overview

Status

Completed

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.

Study Type

Interventional

Enrollment (Anticipated)

43

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • North Central Cancer Treatment Group

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Correlative studies
Given PO

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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.
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Survival
Time Frame: Number of days from registration date to the date of death or last follow-up, assessed up to 5 years
Kaplan-Meier survival curves will be generated to estimate survival.
Number of days from registration date to the date of death or last follow-up, assessed up to 5 years
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
Kaplan-Meier survival curves will be generated to estimate time to progression.
Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years
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
Kaplan-Meier survival curves will be generated to estimate response duration.
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Michelle Elliott, North Central Cancer Treatment Group

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2000

Primary Completion (Actual)

December 1, 2007

Study Registration Dates

First Submitted

May 6, 2001

First Submitted That Met QC Criteria

May 22, 2003

First Posted (Estimate)

May 23, 2003

Study Record Updates

Last Update Posted (Estimate)

October 8, 2013

Last Update Submitted That Met QC Criteria

October 7, 2013

Last Verified

October 1, 2013

More Information

Terms related to this study

Other Study ID Numbers

  • NCI-2012-01853 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
  • U10CA025224 (U.S. NIH Grant/Contract)
  • CDR0000068367
  • NCCTG-N9982
  • N9982 (Other Identifier: CTEP)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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