A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)
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
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.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
Up to 5 years
North Central Cancer Treatment Group
United States: Food and Drug Administration
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