Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases - Full Text View

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00154388

Purpose

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.

Condition	Intervention	Phase
Life Threatening Diseases	Drug: Imatinib mesylate	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study
Official Title:	Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases

Resource links provided by NLM:

Genetics Home Reference related topics: breast cancer

MedlinePlus related topics: Cancer Rare Diseases Soft Tissue Sarcoma Tonsils and Adenoids

Drug Information available for: Imatinib Imatinib mesylate

U.S. FDA Resources

Further study details as provided by Novartis:

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

Estimated Enrollment:	191
Study Start Date:	February 2001

Eligibility

Ages Eligible for Study:	15 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00154388

Sponsors and Collaborators

Novartis

Investigators

Principal Investigator:

Michael Heinrich, MD

Oregon Health and Science University

More Information

No publications provided

Study ID Numbers:	CSTI571B2225
Study First Received:	September 9, 2005
Last Updated:	September 6, 2007
ClinicalTrials.gov Identifier:	NCT00154388 History of Changes
Health Authority:	United States: Food and Drug Administration; European Union: European Medicines Agency; Australia: Department of Health and Ageing Therapeutic Goods Administration; Finland: National Agency for Medicines; 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:

adenocarcinoma
leiomyosarcoma
angiosarcoma
synovial sarcoma
myelodysplastic syndrome/HES
CMML
multiple myeloma
embryonal rhabdomyosarcoma
endometrial sarcoma
adenoid cystic carcinoma
fibromatosis
ductal invasive breast carcinoma
chondrosarcoma
pleural tumor
brenner tumor

ewing sarcoma
round cell tumor
seminoma
thymic carcinoma
malignant melanoma
fibrosarcoma breast
dermatofibrosarcoma protuberans
DFSP
ovarian stromal tumor
osteosarcoma
chorioideal melanoma
hemangiopericytoma
myelofibrosis
liposarcoma
SCLC

Study placed in the following topic categories:

Chronic Myelomonocytic Leukemia
Fibrosarcoma
Sarcoma, Endometrial Stromal
Seminoma
Fibromatosis
Protein Kinase Inhibitors
Sarcoma, Synovial
Preleukemia
Carcinoma, Adenoid Cystic
Lung Neoplasms
Hypereosinophilic Syndrome
Osteogenic Sarcoma
Idiopathic Hypereosinophilic Syndrome
Rhabdomyosarcoma
Dermatofibrosarcoma Protuberans

Leukemia, Myelomonocytic, Chronic
Leukemia, Mast-Cell
Breast Neoplasms
Neurilemmoma
Hemangiopericytoma
Ewing's Sarcoma
Multiple Myeloma
Carcinoma
Imatinib
Carcinoma, Small Cell
Liposarcoma
Malignant Mesenchymal Tumor
Sarcoma
Mastocytosis
Pleural Neoplasms

Additional relevant MeSH terms:

Imatinib
Disease Attributes
Pathologic Processes
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents

Therapeutic Uses
Rare Diseases
Enzyme Inhibitors
Protein Kinase Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 02, 2009