Masitinib in Patients With Mastocytosis With Handicap and Bearing the D816V Mutation
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The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V).
A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
Study Start Date :
Actual Primary Completion Date :
Actual Study Completion Date :
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Patients with one of the following documented mastocytosis:
Smouldering systemic mastocytosis
Indolent systemic mastocytosis with organomegaly
Indolent Systemic Mastocytosis having 2 infiltrated organs (skin and bone-marrow)
Any mastocytosis with in the last 6 months at least 3 anaphylactic shocks or syncops requiring either use of adrenaline or medical assistance
Cutaneous Mastocytosis (CM)
Skin biopsy-documented mastocytosis and evaluable disease based upon:
Histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin biopsy
Missing data (c-kit molecular analysis not done) or documented presence of an activating point mutation in the phosphotransferase domain of c-kit such as D816V c-kit mutation in at least one infiltrated organ (bone marrow or skin)
Refractory to at least one of the symptomatic treatments such as:
Proton pump inhibitor
Other therapies used for the symptomatic care
Handicap defined as at least one of the following handicaps:
pruritus score ≥ 6
number of flushes per week ≥ 7
number of stools per day ≥ 4 ,
number of mictions per day ≥ 8 ,
QLQ-C30 score ≥ 60,
Hamilton score ≥ 10
Patients with one of the following mastocytosis:
Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
Mast cell leukemia (MCL)
Aggressive systemic mastocytosis (ASM)
Patient with a major surgery within 2 weeks prior to study entry
No vulnerable population will be included in this study
Life expectancy < 6 months.
Patient is < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ.
Patient with grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
Patient has a severe and/or uncontrolled medical disease.
Patient has a known diagnosis of human immunodeficiency virus (HIV) infection