NCT04333108

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

Phase 3 Study to Compare Oral Masitinib to Placebo in Treatment of Patients With Smouldering or Indolent Severe Systemic Mastocytosis, Unresponsive to Optimal Symptomatic Treatment

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 140 (estimated)
• Sponsor: AB Science · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell mediator release, unresponsive to optimal symptomatic treatment.

Detailed description

Masitinib is a selective tyrosine kinase inhibitor that modulates mast cell activity via inhibition of c-Kit, Lyn and Fyn kinase signaling pathways. This is a multicenter, randomized, double-blind, placebo-controlled, 2-parallel-group, trial comparing oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell mediator release (also referred to as handicaps), unresponsive to optimal symptomatic treatment. The treatment period is 24 weeks. 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.

Conditions

• Indolent Systemic Mastocytosis

Interventions

Type	Name	Description
DRUG	Masitinib	Masitinib 6 mg/kg/day
OTHER	Placebo	Matching placebo
OTHER	Best Supportive Care	Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglycate, antidepressants, leukotriene antagonists and corticosteroids.

Timeline

• Start date: 2020-07-01
• Primary completion: 2024-12-01
• Completion: 2025-06-01
• First posted: 2020-04-03
• Last updated: 2023-05-06

Locations

17 sites across 8 countries: France, Germany, Netherlands, Poland, Romania, Russia, Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study