- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00015821
Thalidomide in Treating Patients With Myelofibrosis
A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
Study Overview
Status
Conditions
Intervention / Treatment
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
Enrollment (Anticipated)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Minnesota
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Rochester, Minnesota, United States, 55905
- North Central Cancer Treatment Group
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
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
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
Sponsor
Investigators
- Principal Investigator: Michelle Elliott, North Central Cancer Treatment Group
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Bone Marrow Diseases
- Hematologic Diseases
- Myeloproliferative Disorders
- Primary Myelofibrosis
- Physiological Effects of Drugs
- Anti-Infective Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Angiogenesis Inhibitors
- Angiogenesis Modulating Agents
- Growth Substances
- Growth Inhibitors
- Anti-Bacterial Agents
- Leprostatic Agents
- Thalidomide
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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