Systemic mastocytosis MedDRA version: 9.1 Level: LLT Classification code 10042949
Conditions
Interventions
Trade Name: SPRYCEL
Pharmaceutical Form: Coated tablet
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Sponsors
UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) A diagnosis of systemic mastocytosis, according to the WHO criteria; 2) A classification of mastocytosis as either aggressive (presence of C-findings) or smouldering/indolent (presence of B-findings) with severe clinical signs or symptoms unresponsive to antimediator treatment; patients with mastocytosis associated with hematologic non mast cell disease (AHNMD) will be included if the two disorders need to be treated independently, as indicated by the Consensus Guidelines 3) Age >18 years old. Fertile men and women must agree to use a medically acceptable form of contraception during the study; 4) A preserved liver and kidney function as assessed by: a) total bilirubin not exceeding 2.0 times the institutional upper limit of normal (ULN); b) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) not exceeding 2.5 times ULN; c) serum creatinine not exceeding 2.0 times ULN. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1) Pregnancy or breast feeding; 2) Uncontrolled CNS involvement by mastocytosis or other medical disorder that would impair the ability of the subject to receive cytoreductive therapy; 3) Uncontrolled or active significant cardiovascular disease; 4) An altered mental status that would prevent understanding or rendering of informed consent; 5) Clinically significant bleeding from the GI tract within the last 2 months; 6) Current treatment with prednisone at dose >15 mg/day; 7) Current treatment with cytoreductive drug (IFN, 2-CdA or imatinib). Patients can be enrolled in the study if this treatment has been discontinued at least 1 month prior of enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Assessment of the clinical response rate in patients with a diagnosis of systemic mastocytosis in terms of both B/C findings and mediator-related symptoms;Secondary Objective: 1)To assess the duration of response (DOR) and progression free survival (PFS); 2)to evaluate the changes in specific biological markers and molecular changes; 3) to evaluate the safety and toxicity of repeated oral administration of dasatinib. Additional information derived from post-hoc analysis will be: 1) evaluation of efficacy of dasatinib in patients carrying the D816V mutation as compared to that in patients with other mutations or with wild-type KIT and 2) development of an ex vivo system to evaluate therapeutic response to dasatinib.;Primary end point(s): Assessment of the clinical response rate in patients with a diagnosis of systemic mastocytosis in terms of both clincal findings related to organ infiltration by mast cells and mediator-related symptoms | — |
Countries
Italy
Outcome results
None listed