Glivec Phase II Pediatric Study

Study Overview
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Brief Summary
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The purpose of this study is to determine whether Glivec is effective, in children, adolescents and young adults, in the treatment of malignant disease in which evidence suggests a potential pathogenic role of one or more of the tyrosine kinases known to be inhibited by Glivec.

Official Title
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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

Conditions
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Cancer

Intervention / Treatment
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* Drug: Glivec
* Drug: Gleevec


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 patients. National and, when needed, local ethical approval. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Glivec|nan|
|Drug: Gleevec|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| - Tumour Response |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free Survival, overall Survival, safety and tolerability, pharmacokinetic profile and pharmacodynamics of Glivec, pharmacogenetics |  |  |