Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
- Patients ≥ 15 years of age
- Life threatening disease documented by conventional criteria to be resistant to
standard, approved therapy.
- Experimental documentation of functional significance of either Abl, Kit (CD117), or
PDGF-R in the relevant target tissue (preferably on a sample taken within 6 weeks of
- ECOG Performance status of 0, 1, or 2.
- Adequate end organ function defined as: total bilirubin < 1.5 x ULN, SGOT and SGPT <
2.5 x UNL (or < 5 x ULN for patients with hepatic disease), creatinine < 1.5 x ULN,
ANC > 1.5 x 109/L, platelets > 100 x 109/L.
- Negative serum or urine pregnancy test for women of child bearing potential (WOCBP)
within 7 days of study initiation. Post menopausal women must have experienced
amenorrhea for at least 12 months. Male and female patients must use effective birth
control methods throughout the study and for up to 3 months after study
- Life expectancy of more than 3 months.
- Written, voluntary, informed consent for retrieval, evaluation and investigational
use of tissue samples.
- Patients who have received any other investigational agent within 28 days of study
- Patients with another primary malignancy except if other primary malignancy is
neither currently clinically significant nor requiring active intervention.
- Patients with Grade III/IV cardiac problems defined by the New York Heart Association
Criteria (e.g. congestive heart failure, myocardial infarction within 6 months of
- Female patients who are pregnant or breast-feeding.
- Patients who have another severe and/or life threatening medical disease.
- Patients with acute or known chronic liver disease (e.g. chronic active hepatitis,
- Patients with a known diagnosis of the human immunodeficiency virus ((HIV) infection.
- Patients who have received chemotherapy within 4 weeks (6 weeks allowed for
nitrosourea, mitomycin-C or any antibody therapy) prior to study entry.
- Patients who have had major surgery within 2 weeks prior to study entry.
- Patients with any significant history of non-compliance to medical regimens or with
inability to grant reliable informed consent.
Other protocol-defined inclusion/exclusion criteria may apply.