A Phase II Trial of R115777 in Myelofibrosis With Myeloid Metaplasia (MMM)
I. To evaluate the response rate in MMM patients treated with R115777. II. To evaluate the
toxicity of R115777 in patients with MMM.
I. To evaluate the benefit of therapy with R115777 in alleviating disease-associated anemia
in patients with MMM.
II. To evaluate the benefit of R115777 in reducing palpable splenomegaly in patients with
III. To evaluate the effect of R115777 on the hypercatabolic symptoms from MMM. IV. To
evaluate the effect of R115777 on the pathologic increase in circulating myeloid progenitors
in MMM patients through baseline measurement and measurement after the first cycle.
V. To correlate response/relapse with in vitro myeloid colony sensitivity to R115777 at the
time of either response or relapse.
VI. To evaluate the effect of R115777 on bone marrow histologic features of MMM including
osteosclerosis, reticulin fibrosis, and angiogenesis (through serial bone marrow microvessel
OUTLINE: This is a multicenter study.
Patients receive oral tipifarnib twice daily on days 1-21. Treatment repeats every 28 days
for up to 6 courses in the absence of disease progression or unacceptable toxicity.
Patients are followed every 3 months until disease progression and then every 6 months for
up to 2 years.
PROJECTED ACCRUAL: A total of 18-35 patients will be accrued for this study within 15
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Confirmed response defined as the objective status of complete response (CR) or partial response (PR) on 2 consecutive evaluations at least 4 weeks apart
Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Up to 2 years
United States: Food and Drug Administration
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