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

Phase 2

Completed

• Conditions: Mastocytosis
• Interventions: Drug: masitinib

• Registration Number: NCT01266369
• Lead Sponsor: AB Science

Brief 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).

Detailed Description

Not available

Study Timeline

• Study Start: 2007-02
• Primary Completion: 2007-11
• Study Completion: 2010-05
• Study First Submitted: 2010-12-23
• Study First Submitted QC: 2010-12-23
• Study First Posted: 2010-12-24
• Status Verified: 2018-12
• Last Update Submitted: 2018-12-06
• Last Update Posted: 2018-12-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
masitinib 3 mg/kg/day	masitinib	masitinib 3 mg/kg/day
masitinib 6 mg/kg/day	masitinib	masitinib 6 mg/kg/day

Primary Outcome Measures

Name	Time	Method
efficacy on handicaps	week 12	Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12