Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00814073
• Date of registration: 22/12/2008
• Prospective Registration: No
• Primary sponsor: AB Science
• Public title: Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis AB06006
• Scientific title: Randomized, Placebo-controlled, Phase 3 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
• Date of first enrolment: December 2008
• Target sample size: 135
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00814073
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

France, United States

Contacts

• Name: Olivier Lortholary, MD, PhD
• Affiliation: Hôpital Necker, Paris, France

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patient with one of the following documented mastocytosis as per WHO classification:
Smouldering Systemic Mastocytosis, Severe Indolent 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: 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 mictions per day = 8, Fatigue Impact Scale
total score (asthenia) = 75

5. Patients with OPA = 2 (moderate to intolerable general handicap)

6. ECOG = 2

7. Patient with adequate organ function

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 leukemia (MCL), Aggressive systemic mastocytosis (ASM)

2. Previous treatment with any Tyrosine Kinase Inhibitor

3. Patient with recent cardiac history of: Acute coronary syndrome, Acute heart failure,
Significant ventricular arrhythmia; patient with cardiac failure class III or IV;
Syncope without known aetiology within 3 months, uncontrolled severe hypertension.

4. 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

5. Change in the symptomatic treatment of mastocytosis or administration of any new
treatment of mastocytosis within 4 weeks prior to baseline

6. Treatment with any investigational agent within 4 weeks prior to baseline



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Indolent Systemic Mastocytosis

Intervention(s)

Drug: Placebo

Drug: Masitinib

Other: Best Supportive Care

Primary Outcome(s)

Cumulative response (4R75%) [Time Frame: 24 weeks]

Secondary Outcome(s)

Cumulative response (3R75%) [Time Frame: 24 weeks]

Cumulative response (2R75%) [Time Frame: 24 weeks]

Secondary ID(s)

AB06006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors