Clinical trial NCT01137916: Study to Evaluate Imatinib in Desmoid Tumors
Official title: Phase II Study to Evaluate Glivec (Imatinib Mesylate) to Induce Progression Arrest in Aggressive Fibromatosis / Desmoid Tumors Not Amenable to Surgical Resection With R0 Intent or Accompanied by Unacceptable Function Loss
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Aggressive Fibromatosis
Desmoid Tumor
Lead sponsor: Heidelberg University
Sponsor class: OTHER
Collaborators: name: Novartis
First posted: 2010-06-07
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of the present study is to evaluate the activity and safety of imatinib in patients with aggressive fibromatosis who, after receiving the standard therapy, show an inoperable recurrent tumor or disease not readily controllable by surgery or radiotherapy.


Eligibility criteria: Inclusion Criteria:

* Patients with histological confirmed aggressive fibromatosis (desmoid tumor)
* Measurable disease according to the RECIST criteria
* Evidence of relapse or disease progression within the last 6 months (based on RECIST criteria) in computed tomography or magnetic resonance imaging
* No possibility of complete surgical resection or cases in which surgical therapy leaving a large tissue defect, functional deficit or disfigurement would be required
* No possibility of curative radiotherapy with acceptable toxicity and/or late morbidity
* Previous treatment of the tumor region by surgical intervention and/or radiotherapy and/or antihormonal therapy possible
* Age \> or = 18 years
* WHO PS \< or = 1
* Effective contraception during study medication
* Signed informed consent form

Exclusion Criteria:

* Surgical intervention \< 4 weeks
* Prior therapy with imatinib
* Pregnancy or lactation
* Severe hepatic dysfunction
* Known allergic reaction to imatinib or one of its components
* The following laboratory values: Absolute neutrophil count \< 1.5 x 103/mm3, Platelets \< 100,000/mm3, Serum creatinine \> or = 2.5 mg/dl, SGOT and/or SGPT \> 2.5 x ULN (upper limit of normal), Total bilirubin \> 1.5 x ULN
* Participation in another study (four weeks before and during the study)
* Prior malignancy apart from completely resected basal cell carcinoma of the skin or carcinoma in situ of the uterine cervix

Primary outcomes:
measure: Non-progression rate after 6 months of treatment
timeFrame: 6 months
Secondary outcomes:
measure: Non-progression rate after 12 and 24 months of treatment
timeFrame: 12 and 24 months
measure: Response rate
timeFrame: 12 and 24 months
measure: Progression-free survival (PFS) and overall survival (OS)
timeFrame: 12 and 24 months
measure: Recording of patient quality of life
timeFrame: 12 and 24 months
Other outcomes:
Locations:
city: Mannheim
country: Germany
facility: University of Heidelberg, Mannheim University Medical Center
Study officials:
affiliation: University of Heidelberg, Mannheim University Medical Center
name: Bernd Kasper, PD Dr. med.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kasper B, Gruenwald V, Reichardt P, Bauer S, Rauch G, Limprecht R, Sommer M, Dimitrakopoulou-Strauss A, Pilz L, Haller F, Hohenberger P. Imatinib induces sustained progression arrest in RECIST progressive desmoid tumours: Final results of a phase II study of the German Interdisciplinary Sarcoma Group (GISG). Eur J Cancer. 2017 May;76:60-67. doi: 10.1016/j.ejca.2017.02.001. Epub 2017 Mar 8.
citation: Kasper B, Gruenwald V, Reichardt P, Bauer S, Hohenberger P, Haller F. Correlation of CTNNB1 Mutation Status with Progression Arrest Rate in RECIST Progressive Desmoid-Type Fibromatosis Treated with Imatinib: Translational Research Results from a Phase 2 Study of the German Interdisciplinary Sarcoma Group (GISG-01). Ann Surg Oncol. 2016 Jun;23(6):1924-7. doi: 10.1245/s10434-016-5132-4. Epub 2016 Feb 9.