Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment
Indolent Systemic Mastocytosis
About this trial
This is an interventional treatment trial for Indolent Systemic Mastocytosis focused on measuring Mastocytosis with handicap, Mastocytosis, Mast cell, Mast cell infiltration, Skin, Bone marrow, Pruritus, Flushes, c-kit, c-kit mutation, Wild Type, Mutation Asp-816-Val(D816V), Indolent systemic mastocytosis, smoldering systemic mastocytosis
Eligibility Criteria
Inclusion Criteria:
- Patient with one of the following documented mastocytosis subtypes (variants): Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis
- An excess of mast cells or a presence of abnormal mast cells in at least two organs (among skin, bone-marrow and GI Tract).
- Patient with documented systemic mastocytosis and evaluable disease based upon histological criteria
- Patient with documented treatment failure of his/her symptom(s) (within the past 2 years) with at least two of the symptomatic treatments used at optimized dose: Anti H1, Anti H2, Proton pump inhibitor, Antidepressants, Cromoglycate Sodium, Antileukotriene.
- Patient with severe symptoms of mastocytosis over the 14-day run-in period including at least one among pruritus, flushes, and depression: pruritus score ≥ 9, number of flushes per week ≥ 8, Hamilton rating scale for depression (HAMD-17) score ≥ 19.
Exclusion Criteria:
- Patient with one of the following mastocytosis: Cutaneous Mastocytosis, Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD), Mast cell leukemia (MCL), Aggressive systemic mastocytosis (ASM)
- Previous treatment with any Tyrosine Kinase Inhibitor
- Treatment with any investigational agent within 8 weeks prior to screening.
Sites / Locations
- Centre Hospitalier Universitaire d'AmiensRecruiting
- Hospital Jean-MinjozRecruiting
- Grenoble University HospitalRecruiting
- Hospital Claude HuriezRecruiting
- Marseille University Hospital TimoneRecruiting
- Centre de référence de Mastocytose (CEREMAST)Recruiting
- Poitiers University HospitalRecruiting
- Centre Hospitalier UniversitaireRecruiting
- University Hospital Charité
- Erasmus University Medical CenterRecruiting
- Medical University of GdańskRecruiting
- The University Hospital in Krakow (Szpital Uniwersytecki w Krakowie)Recruiting
- University Hospital in Bucharest (Spitalul Universitar de Urgență București)Recruiting
- Almazov National Medical Research CentreRecruiting
- Dnipropetrovsk Clinical Association of Emergency Medical Care of Dnipropetrovsk RegionalRecruiting
- Private Enterprise Private Manufacturing Company AcinusRecruiting
- Guy's and St Thomas' NHS Foundation TrustRecruiting
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Masitinib & BSC
Placebo & BSC
Experimental Arm: Masitinib (titration to 6.0 mg/kg/day) administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control.
Placebo Comparator: Matching placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)