Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment

Launched by AB SCIENCE · Apr 1, 2020

Keywords

ClinConnect Summary

This clinical trial is studying a medication called masitinib to see if it can help people with severe symptoms of indolent or smoldering systemic mastocytosis. This condition involves an excess of certain cells in the body that can cause various uncomfortable symptoms, like itching, flushing, and depression. The goal is to find out if masitinib is more effective than a placebo (a dummy treatment) for patients who have not found relief from their symptoms using other treatments.

To be eligible for this study, participants must have a specific type of mastocytosis and show evidence of having too many abnormal mast cells in at least two organs, such as the skin or digestive system. They should also have tried and failed to manage their symptoms with at least two different treatments in the past two years. During the trial, participants will take the medication or placebo and be monitored for how well it works and any side effects. The trial is currently recruiting patients aged 18 and older, regardless of gender. This is an important step in finding better treatment options for those suffering from this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Patient with one of the following documented mastocytosis subtypes (variants): Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis
• • 2. An excess of mast cells or a presence of abnormal mast cells in at least two organs (among skin, bone-marrow and GI Tract).
• • 3. Patient with documented systemic mastocytosis and evaluable disease based upon histological criteria
• • 4. 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.
• • 5. 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:
• • 1. 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)
• • 2. Previous treatment with any Tyrosine Kinase Inhibitor
• • 3. Treatment with any investigational agent within 8 weeks prior to screening.