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Thalidomide in Treating Patients With Myelofibrosis

Primary Purpose

Primary Myelofibrosis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
thalidomide
laboratory biomarker analysis
Sponsored by
National Cancer Institute (NCI)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Myelofibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • North Central Cancer Treatment Group

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment (thalidomide)

Arm Description

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.

Outcomes

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.
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
Kaplan-Meier survival curves will be generated to estimate survival.
Time to progression
Kaplan-Meier survival curves will be generated to estimate time to progression.
Response duration
Kaplan-Meier survival curves will be generated to estimate response duration.

Full Information

First Posted
May 6, 2001
Last Updated
October 7, 2013
Sponsor
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00015821
Brief Title
Thalidomide in Treating Patients With Myelofibrosis
Official Title
A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2013
Overall Recruitment Status
Completed
Study Start Date
May 2000 (undefined)
Primary Completion Date
December 2007 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Cancer Institute (NCI)

4. Oversight

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Myelofibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
43 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Treatment (thalidomide)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
thalidomide
Intervention Description
Given PO
Intervention Type
Other
Intervention Name(s)
laboratory biomarker analysis
Intervention Description
Correlative studies
Primary Outcome Measure Information:
Title
Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
Description
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.
Time Frame
Up to 5 years
Secondary Outcome Measure Information:
Title
Survival
Description
Kaplan-Meier survival curves will be generated to estimate survival.
Time Frame
Number of days from registration date to the date of death or last follow-up, assessed up to 5 years
Title
Time to progression
Description
Kaplan-Meier survival curves will be generated to estimate 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
Title
Response duration
Description
Kaplan-Meier survival curves will be generated to estimate 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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michelle Elliott
Organizational Affiliation
North Central Cancer Treatment Group
Official's Role
Principal Investigator
Facility Information:
Facility Name
North Central Cancer Treatment Group
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

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Thalidomide in Treating Patients With Myelofibrosis

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