Clinical trial NCT00180830: Glivec Phase II Pediatric Study
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Cancer
Lead sponsor: Gustave Roussy, Cancer Campus, Grand Paris
Sponsor class: OTHER
First posted: 2005-09-16
Enrollment: 36
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: - Tumour Response
Secondary outcomes:
measure: Progression-free Survival, overall Survival, safety and tolerability, pharmacokinetic profile and pharmacodynamics of Glivec, pharmacogenetics
Other outcomes:
Locations:
city: Villejuif
country: France
facility: Institut Gustave-Roussy
city: Amsterdam
country: Netherlands
facility: Emma Kinderziekenhuis AMC
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
Study officials:
affiliation: Gustave Roussy, Cancer Campus, Grand Paris
name: Gilles Vassal, MD,PhD
role: STUDY_CHAIR
affiliation: Birmingham Children's Hospital
name: Bruce Morland
role: STUDY_CHAIR
References: