A 24-week with possible extension, prospective, multicentre, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, Phase III study to compare efficacy and safety of masitinib at 6 mg/kg/day to placebo in treatment of patients with Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis with handicap
- Conditions
- Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis with handicapsMedDRA version: 9.1 Level: LLT Classification code 10026891 Term: Mastocytosis
- Registration Number
- EUCTR2008-000972-25-FR
- Lead Sponsor
- AB SCIENCE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 200
1.Patient with one of the following documented mastocytosis as per WHO classification:
?Smouldering Systemic Mastocytosis
?Indolent Systemic Mastocytosis
?Cutaneous Mastocytosis
2.Patient with documented mastocytosis and evaluable disease based upon histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin and/or bone marrow biopsy
3.Patient with documented treatment failure of his/her handicap(s) with at least one of the following therapy used at optimized dose (refer to table 2):
?Anti H1
?Anti H2
?Proton pump inhibitor
?Osteoclast inhibitor
?Cromoglycate Sodium
?Antileukotriene
4.Handicapped status defined as at least two of the following handicaps, including at least one among pruritus, flushes, depression and asthenia:
?pruritus score = 6
?number of flushes per week = 7
?Hamilton rating scale (depression) = 10
?number of stools per day = 4 ,
?number of mictions per day = 8 ,
?Fatigue Impact Scale total score (asthenia) = 40
5.Patients with OPA > 2 (moderate to intolerable general handicap)
6.ECOG = 2
7.Patient with adequate organ function :
?absolute neutrophil count (ANC) = 2.0 x 109/L,
?platelets (PTL) = 100 x 109/L
?AST/ALT = 2.5x ULN (= 5 x ULN in case of liver mast cell involvement),
?bilirubin = 1.5x ULN
?creatinin = 1.5 x ULN
?albumin > 0.75 x LLN
?urea = 1.5 x ULN
?negative proteinuria defined as negative dipstick; in case of the positive dipstick , 24 hours proteinuria < 1.5g/24 hours
8.Male or female patient with age = 18 years
9.Men and women of child bearing potential (entering the study after a confirmed menstrual period and who have a negative pregnancy test) must agree to use 2 methods of medically acceptable forms of contraception during the study. Men and women of childbearing potential must agree upon using contraception for three months following their participation in the study
10.Patient should be able and willing to comply with study visits and procedures per protocol
11.Patient should understand, sign, and date the written voluntary informed consent form at the screening visit prior to any protocol-specific procedures performed
12.Patient must be covered by insurance for routine costs
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
1.Patient 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)
2.Previous treatment with any Tyrosine Kinase Inhibitor
3.Patient who had major surgery within 2 weeks prior to screening visit
4.Vulnerable population defined as :
?Life expectancy < 6 months
?Patient with < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ
?Patient with grade III/IV cardiac conditions as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
?Patient with any severe and/or uncontrolled medical condition
?Patient with known diagnosis of human immunodeficiency virus (HIV) infection
5.Patient with history of poor compliance or history of drug/alcohol abuse, or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent
6.Patient with any condition that the physician judges could be detrimental to subjects participating in this study; including any clinically important deviations from normal clinical laboratory values or concurrent medical events
Previous treatment
7.Change in the symptomatic treatment of mastocytosis or administration of any new treatment of mastocytosis within 4 weeks prior to baseline
8.Treatment with any investigational agent within 4 weeks prior to baseline
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method