Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases

Life Threatening Diseases Clinical Trial

Official title:

Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00154388
• Other study ID #: CSTI571B2225
• Status: Completed
• Phase: Phase 2
• First received: September 9, 2005
• Last updated: November 16, 2016
• Start date: February 2001
• Est. completion date: January 2007

Study information

• Verified date: November 2016
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationEuropean Union: European Medicines AgencyAustralia: Department of Health and Ageing Therapeutic Goods AdministrationFinland: Finnish Medicines AgencyItaly: Ministry of HealthNetherlands: Medicines Evaluation Board (MEB)United Kingdom: Medicines and Healthcare Products Regulatory AgencyBelgium: Ministry of Social Affairs, Public Health and the Environment
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 185
• Est. completion date: January 2007
• Est. primary completion date: January 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 15 Years to 70 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Life Threatening Diseases
• Rare Diseases

Intervention

Drug:
• Imatinib mesylate

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

References & Publications (1)

Chugh R, Wathen JK, Maki RG, Benjamin RS, Patel SR, Meyers PA, Priebat DA, Reinke DK, Thomas DG, Keohan ML, Samuels BL, Baker LH. Phase II multicenter trial of imatinib in 10 histologic subtypes of sarcoma using a bayesian hierarchical statistical model. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	To identify the contribution of specific protein tyrosine kinases (PTKs) of that specific disease
Secondary	To assess the safety and tolerability of Imatinib mesylate
Secondary	To evaluate the pharmacokinetic profile of Imatinib mesylate
Secondary	To assess, where feasible, the functional significance of relevant signal-transduction components in target tissues

External Links

•   Results for CSTI571B2225 from the Novartis Clinical Trials website