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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 ) Identifier:
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.

Condition Intervention Phase
Life Threatening Diseases
Drug: Imatinib mesylate
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases

Resource links provided by NLM:

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

Enrollment: 185
Study Start Date: February 2001
Study Completion Date: January 2007
Primary Completion Date: January 2007 (Final data collection date for primary outcome measure)

Ages Eligible for Study:   15 Years to 70 Years   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00154388

Sponsors and Collaborators
Novartis Pharmaceuticals
Principal Investigator: Michael Heinrich, MD Oregon Health and Science University
  More Information

Responsible Party: Novartis Pharmaceuticals Identifier: NCT00154388     History of Changes
Other Study ID Numbers: CSTI571B2225 
Study First Received: September 9, 2005
Last Updated: April 18, 2012
Health Authority: 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:
dermatofibrosarcoma protuberans
synovial sarcoma
myelodysplastic syndrome/HES
multiple myeloma
embryonal rhabdomyosarcoma
endometrial sarcoma
adenoid cystic carcinoma
ductal invasive breast carcinoma
pleural tumor
brenner tumor
ewing sarcoma
round cell tumor
thymic carcinoma
malignant melanoma
fibrosarcoma breast
ovarian stromal tumor
chorioideal melanoma

Additional relevant MeSH terms:
Rare Diseases
Disease Attributes
Pathologic Processes
Imatinib Mesylate
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on October 27, 2016