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

Phase 1

• Conditions: Smouldering Systemic or Indolent Systemic Mastocytosis with handicaps; MedDRA version: 17.1 Level: PT Classification code 10026891 Term: Mastocytosis System Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2008-000972-25-ES
• Lead Sponsor: AB Science

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-10-03
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 150

Inclusion Criteria

1. Patient with one of the following documented mastocytosis as per WHO classification:
Smouldering Systemic Mastocytosis
Indolent Systemic 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 fatigue:
Pruritus score ? 9
Number of flushes per week ? 8
Hamilton rating scale for depression(HAMD-17) score ? 19
Number of stools per day ? 4
Number of micturition per day ? 8
Fatigue Impact Scale total score (asthenia) ? 75
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 130
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1.Patient with one of the following mastocytosis:
?Cutaneous Mastocytosis
?Not documented Smouldering Systemic Mastocytosis or Indolent Systemic Mastocytosis
?Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
?Mast cell leukaemia (MCL)
?Aggressive systemic mastocytosis (ASM)
2.Previous treatment with any Tyrosine Kinase Inhibitor
3.Patient presenting with cardiac disorders defined by at least one of the following conditions:
?Patient with recent cardiac history (within 6 months) of:
-Acute coronary syndrome
-Acute heart failure (class III or IV of the NYHA classification)
-Significant ventricular arrhythmia (persistent ventricular tachycardia, ventricular fibrillation, resuscitated sudden death)
?Patient with cardiac failure class III or IV of the NYHA classification
?Patient with severe conduction disorders which are not prevented by permanent pacing (atrio-ventricular block 2 and 3, sino-atrial block)
?Syncope without known aetiology within 3 months
?Uncontrolled severe hypertension, according to the judgment of the investigator, or symptomatic hypertension
4.Patient who had major surgery within 2 weeks prior to screening visit
5.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 any severe and/or uncontrolled medical condition
?Patient with known diagnosis of human immunodeficiency virus (HIV) infection
6.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, or institutionalized by court decision
7.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
8.Change in the symptomatic treatment of mastocytosis or administration of any new treatment of mastocytosis within 4 weeks prior to baseline
9.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