Open Label, Pilot Phase II Study of Glivec in Children and Adolescents With Life Threatening Diseases Known to Be Associated With One or More Glivec-Sensitive Tyrosine Kinases
- Patients from 6 months to 21 years of age.
- Malignant disease documented by conventional criteria to be refractory to standard,
approved therapy, or for which no conventional therapies of definitive benefit exist.
- Immunohistochemistry documentation of positivity of either Kit (CD117) or PDGF-R in
tumor tissue relevant. Each positive tumor will be centrally reviewed before
inclusion of the patient in the trial.
- Measurable or evaluable disease.
- WHO Performance status 0,1, or 2 or Lansky Play Scale >= 50%.
- Adequate organ function, defined as the following: total bilirubin < 1.5 x ULN, SGOT
and SGPT < 2.5 x UNL (or < 5 x ULN if hepatic disease involvement is present),
creatinine < 1.5 x ULN, ANC > 1x 109/L, platelets > 75 x 109/L.
- Female patients of child-bearing potential must have negative pregnancy test within 7
days before initiation of study drug dosing.
- Male and female patients of reproductive potential must agree to employ an effective
barrier method of birth control throughout the study and for up to 3 months following
discontinuation of study drug.
- Life expectancy of more than 6 weeks.
- Written, voluntary, informed consent, including consent for retrieval and
investigational use of tissue samples for evaluation signed by parents or young adult
- National and, when needed, local ethical approval.
- Patient with hematological disease positive for the chimeric BCR-ABL fusion protein
or for c-kit.
- Patient has received any other investigational agents within 28 days of first day of
study drug dosing.
- Female patients who are pregnant or breast-feeding.
- Patient has another severe and/or life-threatening medical diseasePatient has an
acute or known chronic liver disease (e.g., chronic active hepatitis, cirrhosis).
- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
- Patient has received chemotherapy within 4 weeks (6 weeks for nitrosourea,
mitomycin-C or any antibody therapy) prior to study entry unless urgent enrollment
needed and approved by the study coordinator.