Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
First received: September 9, 2005
Last updated: April 18, 2012
Last verified: April 2012
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of specific protein tyrosine kinases (PTKs) of that specific disease.
Life Threatening Diseases
Drug: Imatinib mesylate
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
||Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Primary Outcome Measures:
- To examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate-sensitive tyrosine kinases
- To identify the contribution of specific protein tyrosine kinases (PTKs) of that specific disease
Secondary Outcome Measures:
- To assess the safety and tolerability of Imatinib mesylate
- To evaluate the pharmacokinetic profile of Imatinib mesylate
- To assess, where feasible, the functional significance of relevant signal-transduction components in target tissues
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||January 2007 (Final data collection date for primary outcome measure)
|Ages Eligible for Study:
||15 Years to 70 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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 study entry).
- 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 discontinuation.
- 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 initiation.
- 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 study).
- 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, cirrhosis).
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00154388
||Michael Heinrich, MD
||Oregon Health and Science University
No publications provided
||Novartis ( Novartis Pharmaceuticals )
History of Changes
|Other Study ID Numbers:
|Study First Received:
||September 9, 2005
||April 18, 2012
||United States: Food and Drug Administration
European Union: European Medicines Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration
Finland: Finnish Medicines Agency
Italy: Ministry of Health
Netherlands: Medicines Evaluation Board (MEB)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Belgium: Ministry of Social Affairs, Public Health and the Environment
Keywords provided by Novartis:
adenoid cystic carcinoma
ductal invasive breast carcinoma
round cell tumor
ovarian stromal tumor
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on December 03, 2013
Protein Kinase Inhibitors
Molecular Mechanisms of Pharmacological Action