Protein-Tyrosine Kinase Inhibitor (STI571) for Treatment of Patients With Ph+ Chronic Myeloid Leukemia
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
This is a Phase II, open-label, multi-center trial designed primarily to evaluate the rate of
complete or major cytogenetic response of STI571 as demonstrated by a decrease in the
percentage of Ph chromosome positive cells in the bone marrow, in patients with CML who are
refractory to or intolerant of interferon-alpha.
During the core phase of the study, patients will receive once daily oral administration of
STI571 at a dose of 400 mg, for up to 12 months. After completing 12 months of therapy
patients may be eligible to receive additional therapy provided that, in the opinion of the
investigator, the patient has benefited from treatment with STI571 and in the absence of
safety concerns. Patients will receive STI571 on an outpatient basis.
During the extended phase (which is of indefinite duration), patients may continue STI571
until either progression to accelerated phase, blast phase, death, the development of
intolerable toxicity, or the investigator feels it is no longer in the patient's best
interest to continue therapy, whichever comes first. The number of visits will be at a
reduced frequency. Patients who discontinue study drug will be followed for survival for up
to 5 years.
STI571 will be considered active if the interferon-refractory patient population satisfies
the target of achieving a complete or major response at a rate of at least 30%, within the
preset error limits. Cytogenetic responses will be evaluated every three months and
categorized as either complete (0% Ph+ chromosome cells), or major (1 to 35% Ph+ chromosome
cells) responses. STI571 will be discontinued for any patient whose disease progresses to
either the accelerated phase or blast crisis.
A minimum of 100 patients who are interferon refractory will receive STI571 administered at a
dose of 400 mg once a day. In addition, the protocol is also open for patients who are
intolerant to interferon-alpha in order to get a preliminary evaluation of their response to
STI571 therapy. Up to 100 intolerant patients will be enrolled. Enrollment of intolerant
patients will cease at 100, or whenever the 100 refractory patients are accrued, whichever
comes first.