Clinical trial NCT00287846: Imatinib Mesylate in Treating Patients With Recurrent or Refractory Fibromatosis
Official title: Multicentric Phase I/II Study Evaluating the Efficacy and Toxicity of Imatinib in Adult Patients With Aggressive Fibromatosis That Cannot be Treated by Surgery or Curative Radiotherapy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Desmoid Tumor
Keywords: desmoid tumor
Lead sponsor: UNICANCER
Sponsor class: OTHER
First posted: 2006-02-07
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 120
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

PURPOSE: This phase I/II trial is studying the side effects of imatinib mesylate and to see how well it works in treating patients with recurrent or refractory aggressive fibromatosis.

Detailed description: OBJECTIVES:

Primary

* Determine the non-progression rate in patients with recurrent or refractory aggressive fibromatosis after 3 months of treatment with imatinib mesylate.

Secondary

* Determine the non-progression rate in patients after being treated with this drug for 12 months.
* Determine the toxic effects of this drug in these patients.
* Determine the tolerance to this drug in these patients.
* Determine the response rate in patients treated with this drug
* Determine progression free and overall survival of patients treated with this drug.
* Determine the quality of life of patients treated with this drug.
* Correlate clinical, biological, and genomic markers with response and long-term stable disease in patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive oral imatinib mesylate once daily for up to 12 months in the absence of disease progression or unacceptable toxicity.

Quality of life is assessed periodically.

PROJECTED ACCRUAL: A total of 39 patients will be accrued for this study.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed aggressive fibromatosis (desmoid tumor)
* Relapse or disease progression despite surgery, chemotherapy, radiotherapy, or any other treatment
* Tumors must meet the following criteria:

  * Ineligible for complete surgical resection by carcinological exeresis OR surgery would cause severe mutilation
  * Cannot be treated with curative radiotherapy
* Measurable disease by RECIST criteria
* No prior malignancy

PATIENT CHARACTERISTICS:

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 6 months after completion of study treatment
* Absolute neutrophil count \> 1,000/mm\^3
* Platelet count \> 100,000/mm\^3
* Bilirubin \< 1.5 times upper limit of normal (ULN)
* SGOT and SGPT \< 2.5 times ULN
* Creatinine ≤ 2.5 times normal
* No severe liver failure
* No chronic somatic or psychiatric illness that would preclude study compliance
* No known hypersensitivity to imatinib mesylate or one of its components
* No geographical, social, or psychological reason that would inhibit follow-up

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* No concurrent immunomodulators\*
* No concurrent hormonal treatments\* if used for fibromatosis
* No concurrent cytotoxic drugs\*
* No concurrent nonsteroidal anti-inflammatory drug\* if used for fibromatosis

  * Allowed if used as an analgesic 3 months prior to disease progression
* No concurrent participation in another therapeutic investigational trial NOTE: \*If disease progression has occurred during this treatment, then the treatment must have ended ≥ 1 month prior to study entry

Primary outcomes:
measure: Non-progression rate
timeFrame: 3 months
Secondary outcomes:
measure: Non-progression rate
timeFrame: 12 months
measure: Toxic effects
timeFrame: 12 months
measure: Tolerance
timeFrame: 12 months
measure: Response rate
timeFrame: 5 years
measure: Progression-free survival
timeFrame: the time between the inclusion date and the progression date
measure: Overall survival
timeFrame: the time between the inclusion date and the death whathever the cause
measure: Quality of life
timeFrame: 5 years
measure: Correlation of clinical, biological, and genomic markers with response and long-term stable disease
timeFrame: 5 years
Other outcomes:
Locations:
city: Angers
country: France
facility: Centre Paul Papin
city: Besancon
country: France
facility: Centre Hospitalier Regional de Besancon - Hopital Jean Minjoz
city: Bordeaux
country: France
facility: Institut Bergonie
city: Caen
country: France
facility: Centre Regional Francois Baclesse
city: Lille
country: France
facility: Centre Oscar Lambret
city: Lyon
country: France
facility: Centre Leon Berard
city: Lyon
country: France
facility: Hopital Edouard Herriot - Lyon
city: Marseille
country: France
facility: CHU de la Timone
city: Montpellier
country: France
facility: Centre Regional de Lutte Contre le Cancer - Centre Val d'Aurelle
city: Nantes-Saint Herblain
country: France
facility: CRLCC Nantes - Atlantique
city: Paris
country: France
facility: Institut Curie Hopital
city: Paris
country: France
facility: Hopital Tenon
city: Reims
country: France
facility: Institut Jean Godinot
city: Rennes
country: France
facility: Centre Eugene Marquis
city: Rouen
country: France
facility: Centre Henri Becquerel
city: Saint Cloud
country: France
facility: Centre Rene Huguenin
city: Strasbourg
country: France
facility: Centre Paul Strauss
city: Strasbourg
country: France
facility: Hopitaux Universitaire de Strasbourg
city: Suresnes
country: France
facility: Hopital Foch
city: Toulouse
country: France
facility: Institut Claudius Regaud
city: Tours
country: France
facility: Centre Hospitalier Universitaire Bretonneau de Tours
city: Vandoeuvre-les-Nancy
country: France
facility: Centre Alexis Vautrin
city: Villejuif
country: France
facility: Institut Gustave Roussy
Study officials:
affiliation: Hopital Edouard Herriot - Lyon
name: Jean-Yves Blay, MD, PhD
role: STUDY_CHAIR
References:
citation: Penel N, Le Cesne A, Bui BN, Perol D, Brain EG, Ray-Coquard I, Guillemet C, Chevreau C, Cupissol D, Chabaud S, Jimenez M, Duffaud F, Piperno-Neumann S, Mignot L, Blay JY. Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up. Ann Oncol. 2011 Feb;22(2):452-7. doi: 10.1093/annonc/mdq341. Epub 2010 Jul 9.
citation: Fayette J, Dufresne A, Penel N, et al.: Imatinib for the treatment of aggressive fibromatosis/desmoid tumors (AF/DT) failing local treatment: updated outcome and predictive factors for progression free survival: a FNCLCC French Sarcoma Group-GETO study. [Abstract] J Clin Oncol 25 (Suppl 18): A-10062, 560s, 2007.