NCT05449444

Masitinib for the Treatment of Severe Mast Cell Activation Syndrome

A 24-week, Multicenter, Randomized, Double Blind, Placebo-controlled, Dose-range Finding Phase II Study to Compare Efficacy and Safety of Oral Masitinib to Placebo in Treatment of Patients With Severe Mast Cell Activation Syndrome (MCAS) With Handicap Unresponsive to Optimal Symptomatic Treatment

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 72 (estimated)
• Sponsor: AB Science · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the efficacy and safety of two dosing schemes of oral masitinib versus matching placebo in the treatment of patients suffering from severe MCAS with handicap unresponsive to optimal symptomatic treatment.

Detailed description

Multicenter, double-blind, placebo-controlled trial comparing two different dosing schemes over a 24-week treatment period. Dosing scheme #1: Oral masitinib treatment at 3 mg/kg/day for 4 weeks, then a switch to 4.5 mg/kg/day for the remainder of the treatment period, versus placebo with a matching titration scheme. Randomization 2: 1 (Masitinib MCAS: Placebo MCAS). Dosing scheme #2: Oral masitinib treatment at 3 mg/kg/day for 4 weeks, then a switch to 4.5 mg/kg/day for 4 weeks, then a second switch to 6 mg/kg/day for the remainder of the treatment period versus placebo treatment with a matching titration scheme. Randomization 2: 1 (Masitinib MCAS: Placebo MCAS)

Conditions

• Mast Cell Activation Syndrome

Interventions

Type	Name	Description
DRUG	Masitinib 4.5 mg/kg/day	Masitinib 4.5 mg/kg/day
DRUG	Placebo	Matching placebo
OTHER	Best supportive care	Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.
DRUG	Masitinib 6.0 mg/kg/day	Masitinib 6.0 mg/kg/day

Timeline

• Start date: 2022-07-01
• Primary completion: 2024-12-31
• Completion: 2024-12-31
• First posted: 2022-07-08
• Last updated: 2023-02-06

Locations

4 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study