Masitinib in Patients With Mastocytosis With Handicap and Bearing the D816V Mutation

• Status: Completed
• Trial end date: 2010-05-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AB Science

Criteria

Inclusion Criteria:

1. 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)

2. 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

• Clinical criteria: typical skin lesions (maculopapular, urticaria pigmentosa, mastocytoma)

3. 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)

4. Refractory to at least one of the symptomatic treatments such as:

• Anti H1

• Anti H2

• Proton pump inhibitor

• Osteoclast inhibitor

• Cromoglycate Sodium

• Antileukotriene

• Other therapies used for the symptomatic care

5. 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

Exclusion Criteria:

1. 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)

2. Patient with a major surgery within 2 weeks prior to study entry

3. 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