A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
18 Years
N/A
Both
Primary Myelofibrosis
Trial Information
A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
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.
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
Type of Study:
Interventional
Study Design:
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart.
Outcome 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.
Outcome Time Frame:
Up to 5 years
Safety Issue:
No
Principal Investigator
Michelle Elliott
Investigator Role:
Principal Investigator
Investigator Affiliation:
North Central Cancer Treatment Group
Authority:
United States: Food and Drug Administration
Study ID:
NCI-2012-01853
NCT ID:
NCT00015821
Start Date:
January 2001
Completion Date:
Related Keywords:
- Primary Myelofibrosis
- Primary Myelofibrosis
Name | Location |
|---|---|
| North Central Cancer Treatment Group | Rochester, Minnesota 55905 |
