Study to compare efficacy and safety of masitinib at 6 mg/kg/day to placebo in treatment of patients with Indolent with handicap

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2008-000972-25-CZ

Enrollment

200

Registered

2009-12-01

Start date

2010-01-13

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis with handicaps MedDRA version: 20.0 Level: PT Classification code 10026891 Term: Mastocytosis System Organ Class: 10005329 - Blood and lymphatic system disorders

Interventions

Product Name: AB1010 Tablets Product Code: AB1010 Tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Masitinib mesylate CAS Number: 790-299-79-5 Current Sponsor code: AB1010 Concentr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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 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.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

Design outcomes

Primary

Measure	Time frame
Main Objective: The objective is to compare the safety and efficacy of masitinib to placebo in patients with indolent mastocytosis with handicap. Primary endpoint: ? Responder rate at week 24 ;Secondary Objective: Secondary endpoints: ? Response in at least two out of three variables among pruritus, flushes, depression or asthenia, at week 12 and week 24 ? Responder rate at week 12 ? Pruritus score at week 12 and week 24 ? Number of flushes per week at week 12 and week 24 ? Hamilton score at week 12 and week 24 ? Fatigue Impact Scale at week 12 and week 24 ? Number of mictions per day at week 12 and week 24 ? Number of stools per day at week 12 and week 24 ? QLQ-C30 scores at week 12 and week 24 ? Overall Patient Assessment (OPA) at week 12 and week 24 ? AFIRMM V2 score at week 12 and week 24 ? Percentage of patient without handicap at week 12 and 24 ? Mast cell infiltration in the skin or bone marrow at week 24 ? Serum tryptase level at week 24 Safety profile of masitinib: Occurrence of Adverse Events, vital signs, EKG, Chest X-Ray, cardiac ultrasonography and biological parameters. ;Primary end point(s): Primary variable: Responder rate A patient is classified as responder if he/she presents an improvement of = 50% in at least one handicap, handicap being defined as a score above the cut in any of the 4 variables: - pruritus score = 6, - number of flushes per week = 7, - Hamilton score = 10, - Fatigue Impact Scale score = 40. Any patient with a worsening of more than 50% of any handicap, and/or with emergence of a new handicap with an increase of more than 50% from baseline, will be considered a non responder ;Timepoint(s) of evaluation of this end point: Week 24	—

Measure

Time frame

Main Objective: The objective is to compare the safety and efficacy of masitinib to placebo in patients with indolent mastocytosis with handicap. Primary endpoint: ? Responder rate at week 24 ;Secondary Objective: Secondary endpoints: ? Response in at least two out of three variables among pruritus, flushes, depression or asthenia, at week 12 and week 24 ? Responder rate at week 12 ? Pruritus score at week 12 and week 24 ? Number of flushes per week at week 12 and week 24 ? Hamilton score at week 12 and week 24 ? Fatigue Impact Scale at week 12 and week 24 ? Number of mictions per day at week 12 and week 24 ? Number of stools per day at week 12 and week 24 ? QLQ-C30 scores at week 12 and week 24 ? Overall Patient Assessment (OPA) at week 12 and week 24 ? AFIRMM V2 score at week 12 and week 24 ? Percentage of patient without handicap at week 12 and 24 ? Mast cell infiltration in the skin or bone marrow at week 24 ? Serum tryptase level at week 24 Safety profile of masitinib: Occurrence of Adverse Events, vital signs, EKG, Chest X-Ray, cardiac ultrasonography and biological parameters. ;Primary end point(s): Primary variable: Responder rate A patient is classified as responder if he/she presents an improvement of = 50% in at least one handicap, handicap being defined as a score above the cut in any of the 4 variables: - pruritus score = 6, - number of flushes per week = 7, - Hamilton score = 10, - Fatigue Impact Scale score = 40. Any patient with a worsening of more than 50% of any handicap, and/or with emergence of a new handicap with an increase of more than 50% from baseline, will be considered a non responder ;Timepoint(s) of evaluation of this end point: Week 24

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Secondary

Measure	Time frame
Secondary end point(s): - Response in at least two out of three variables among pruritus, flushes, depression or asthenia, at week 12 and week 24 - Responder rate at week 12 - Pruritus score at week 12 and week 24 - Number of flushes per week at week 12 and week 24 - Hamilton rating scale for depression score at week 12 and week 24 - Fatigue Impact Scale at week 12 and week 24 - Number of micturitions per day at week 12 and week 24 - Number of stools per day at week 12 and week 24 - QLQ-C30 scores at week 12 and week 24 - Overall Patient Assessment (OPA) at week 12 and week 24 - AFIRMM V2 score at week 12 and week 24 - Percentage of patient without handicap at week 12 and 24 - Mast cell infiltration in the skin or bone marrow at week 24 - Serum tryptase level at week 24;Timepoint(s) of evaluation of this end point: Week 12 and 24	—

Measure

Time frame

Secondary end point(s): - Response in at least two out of three variables among pruritus, flushes, depression or asthenia, at week 12 and week 24 - Responder rate at week 12 - Pruritus score at week 12 and week 24 - Number of flushes per week at week 12 and week 24 - Hamilton rating scale for depression score at week 12 and week 24 - Fatigue Impact Scale at week 12 and week 24 - Number of micturitions per day at week 12 and week 24 - Number of stools per day at week 12 and week 24 - QLQ-C30 scores at week 12 and week 24 - Overall Patient Assessment (OPA) at week 12 and week 24 - AFIRMM V2 score at week 12 and week 24 - Percentage of patient without handicap at week 12 and 24 - Mast cell infiltration in the skin or bone marrow at week 24 - Serum tryptase level at week 24;Timepoint(s) of evaluation of this end point: Week 12 and 24

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Countries

Austria, Bulgaria, Czech Republic, France, Germany, Greece, Hungary, India, Italy, Latvia, Poland, Russian Federation, Slovakia, Spain, Ukraine, United Kingdom, United States

Contacts

Public ContactClinical Project Director

AB Science

paul.gineste@ab-science.com33147206240

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026