Patients with NF1 and plessiform neurofibromas MedDRA version: 14.1 Level: SOC Classification code 10029104 Term: Neoplasms benign, malignant and unspecified (incl cysts and polyps) System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Trade Name: GLIVEC*24CPS 100MG
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: IMATINIB
CAS Number: 152459-95-5
Current Sponsor code: NA
Other descriptive name: NA
Concentration unit: mg milli
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: clinical diagnosis of NF1 according US National Institute of Health Consensus criteria, age at entry >3 10 years) performance levels > 50. Radiographic progression is not a necessity for patient entry. Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients undergoing radiation,chemotherapy, hormonal therapy, directed to the tumor or immunotherapy were excluded from partecipation. Other exclusion criteria are the presence of an active optic glioma or other tumor requiring radiation or chemotherapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary aim of this proposal is to verify modification in the rating neurological scale adopted and to estimate progression free survival (according to an automated volumetric MRI analysis) in patients with not total resectable, disabling or disfiguring plexiform fibromas (see inclusion criteria) expressing c-KIT and treated with Imatinib mesylate.;Secondary Objective: Secondary aims include: 1) the evaluation of toxicity during the treatment graded according to NCI CTCAE v 3.0 2) to assess whether tumor specimens harbouring microdeletion of the NF1 gene (constitutional or aquired) show specific characteristics which can be correlated with the patients' response to the treatment; thus allowing the setting of more tailored treatment of NF1 microdeleted patiens that; despite beeing a minority of all NF1 patients, has an enhanced risk to develop tumors (De Raedt et al., 2003); 3) to analyze constitutional DNA mutation in order to assess whether there are correlations between genotype, the risk of developing large neurofibromas, and tumor rensponse to drug; 4) to assess whether previously described increase in the serum levels of SCF.;Primary end point(s): Proportion of patients with stable tumors volume.;Timepoint(s) of evaluation of this end point: 3, 6 and 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - tossicity - modification of neurological and quality of life scale - proportion of patiens with microdeletion of NF1 gene - SCF level in patiens;Timepoint(s) of evaluation of this end point: 3, 6 and 12 months | — |
Countries
Italy
Contacts
Public ContactServizio Ricerca e Sviluppo Clinico
Fondazione Irccs Istituto Neurologico Carlo Besta
Outcome results
None listed