Masitinib for Severe Mast Cell Activation Syndrome

Recruiting in Palo Alto (17 mi)

+3 other locations

Overseen byJulien Rossignol, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: AB Science

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?This trial tests if masitinib, a pill, can help people with severe MCAS who haven't improved with other treatments. It works by calming down cells that cause allergic reactions and inflammation.

Eligibility Criteria

This trial is for patients with severe Mast Cell Activation Syndrome (MCAS) who haven't responded to other treatments. They must have had significant symptoms like intense itching, frequent flushes, or depression during a two-week period and failed at least two optimized dose treatments in the past two years. Participants should be on a stable anti-histamine regimen and not pregnant or breastfeeding.

Inclusion Criteria

I have been on a stable dose of Anti-H1 medication for at least 4 weeks.

I have severe itching, frequent hot flashes, or significant depression.

I've tried at least two treatments for my condition without success in the last two years.

+1 more

Exclusion Criteria

I have a slow-growing form of mastocytosis affecting my whole body.

I have been treated with a Tyrosine Kinase Inhibitor before.

I haven't changed my MCAS treatment or started any new one in the last 4 weeks.

+1 more

Participant Groups

The trial tests how effective and safe different doses of oral masitinib are compared to a placebo in treating severe MCAS unresponsive to optimal symptomatic treatment. Patients will receive either masitinib at 4.5 mg/kg/day, masitinib at 6.0 mg/kg/day, placebo, or best supportive care.

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Masitinib (6.0) & BSCExperimental Treatment2 Interventions

Masitinib 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for4 weeks of treatment, then a second dose escalation to 6 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.

Group II: Masitinib (4.5) & BSCExperimental Treatment2 Interventions

Masitinib 4.5 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.

Group III: Placebo & BSCPlacebo Group2 Interventions

Placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive a matched dose placebo, given orally twice daily.

Masitinib is already approved in European Union, United States for the following indications:

🇪🇺 Approved in European Union as Masivet for: